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Chemistry

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Also known as: 38398-32-2, Ccd 1042, Ztalmy, Ccd-1042, 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one, 98wi44ohiq
Molecular Formula
C22H36O2
Molecular Weight
332.5  g/mol
InChI Key
PGTVWKLGGCQMBR-FLBATMFCSA-N
FDA UNII
98WI44OHIQ

Ganaxolone
Ganaxolone is an orally bioavailable synthetic analog of the neuroactive steroid allopregnanolone and positive allosteric modulator of the gamma-aminobutyric acid (GABA)-A receptor, with anxiolytic, sedative, antidepressant and anticonvulsant activities. Upon oral administration, ganaxolone specifically targets and binds to the allosteric sites of the synaptic and extrasynaptic GABA-A receptor-chloride ionophore complex in the central nervous system (CNS). This leads to an increase in the opening of chloride channels, membrane hyperpolarization, increases the inhibitory effect of GABA on the CNS, and inhibits neurotransmission. This blocks seizure propagation and elevates seizure thresholds.
1 2D Structure

Ganaxolone

2 Identification
2.1 Computed Descriptors
2.1.1 IUPAC Name
1-[(3R,5S,8R,9S,10S,13S,14S,17S)-3-hydroxy-3,10,13-trimethyl-1,2,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydrocyclopenta[a]phenanthren-17-yl]ethanone
2.1.2 InChI
InChI=1S/C22H36O2/c1-14(23)17-7-8-18-16-6-5-15-13-20(2,24)11-12-21(15,3)19(16)9-10-22(17,18)4/h15-19,24H,5-13H2,1-4H3/t15-,16-,17+,18-,19-,20+,21-,22+/m0/s1
2.1.3 InChI Key
PGTVWKLGGCQMBR-FLBATMFCSA-N
2.1.4 Canonical SMILES
CC(=O)C1CCC2C1(CCC3C2CCC4C3(CCC(C4)(C)O)C)C
2.1.5 Isomeric SMILES
CC(=O)[C@H]1CC[C@@H]2[C@@]1(CC[C@H]3[C@H]2CC[C@@H]4[C@@]3(CC[C@@](C4)(C)O)C)C
2.2 Other Identifiers
2.2.1 UNII
98WI44OHIQ
2.3 Synonyms
2.3.1 MeSH Synonyms

1. 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one

2. Ccd 1042

3. Ccd-1042

4. Ztalmy

2.3.2 Depositor-Supplied Synonyms

1. 38398-32-2

2. Ccd 1042

3. Ztalmy

4. Ccd-1042

5. 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one

6. 98wi44ohiq

7. 1-((3r,5s,8r,9s,10s,13s,14s,17s)-3-hydroxy-3,10,13-trimethylhexadecahydro-1h-cyclopenta[a]phenanthren-17-yl)ethanone

8. 1-[(3r,5s,8r,9s,10s,13s,14s,17s)-3-hydroxy-3,10,13-trimethyl-1,2,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydrocyclopenta[a]phenanthren-17-yl]ethanone

9. Pregnan-20-one, 3-hydroxy-3-methyl-, (3a,5a)-

10. Ncgc00165802-02

11. Dsstox_cid_26503

12. Dsstox_rid_81672

13. Dsstox_gsid_46503

14. 1-((3r,5s,8r,9s,10s,13s,14s,17s)-3-hydroxy-3,10,13-trimethylhexadecahydro-1h-cyclopenta[a]phenanthren-17-yl)ethan-1-one

15. Cas-38398-32-2

16. Ganaxolone [usan]

17. Ganaxolone [usan:inn]

18. Unii-98wi44ohiq

19. Ganaxolone, Solid

20. Ganaxolone [mi]

21. Ganaxolone [inn]

22. Ganaxolone (usan/inn)

23. Ganaxolone [who-dd]

24. Schembl144522

25. (3alpha,5alpha)-3-hydroxy-3-methylpregnan-20-one

26. Chembl1568698

27. Dtxsid6046503

28. Gtpl11942

29. Ccd1042

30. Chebi:177658

31. Dea No. 2401

32. Hms3261k19

33. Amy38916

34. Zinc3824281

35. Tox21_112264

36. Tox21_500379

37. Bdbm50369240

38. Mfcd09971088

39. Akos027327586

40. Tox21_112264_1

41. Ccg-221683

42. Db05087

43. Lp00379

44. Sdccgsbi-0633706.p001

45. Ncgc00165802-03

46. Ncgc00261064-01

47. Ncgc00263545-01

48. As-35253

49. B7092

50. (3?,5?)-3-hydroxy-3-methyl-pregnan-20-one

51. 3alpha-hydroxy-3-methyl-5alpha-pregnan-20-one

52. D04300

53. A901692

54. (3alpha,5alpha)-3-hydroxy-3-methyl-pregnan-20-one

55. Q3758034

56. 3.alpha.-hydroxy-3-methyl-5.alpha.-pregnan-20-one

57. Pregnan-20-one, 3-hydroxy-3-methyl-, (3alpha,5alpha)-

58. Pregnan-20-one, 3-hydroxy-3-methyl-, (3.alpha.,5.alpha.)-

59. 1-[(3r,5s,8r,9s,10s,13s,14s,17s)-3-hydroxy-3,10,13-trimethyl-1,2,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydrocyclopenta[a]phenanthren-17-yl]ethanone.

2.4 Create Date
2006-07-28
3 Chemical and Physical Properties
Molecular Weight 332.5 g/mol
Molecular Formula C22H36O2
XLogP35
Hydrogen Bond Donor Count1
Hydrogen Bond Acceptor Count2
Rotatable Bond Count1
Exact Mass332.271530387 g/mol
Monoisotopic Mass332.271530387 g/mol
Topological Polar Surface Area37.3 Ų
Heavy Atom Count24
Formal Charge0
Complexity542
Isotope Atom Count0
Defined Atom Stereocenter Count8
Undefined Atom Stereocenter Count0
Defined Bond Stereocenter Count0
Undefined Bond Stereocenter Count0
Covalently Bonded Unit Count1
4 Drug and Medication Information
4.1 Drug Indication

Investigated for use/treatment in pediatric indications and seizure disorders.


Treatment of cyclin-dependent kinase-like 5 deficiency disorder


5 Pharmacology and Biochemistry
5.1 Pharmacology

Ganaxolone is a powerful positive allosteric modulator of GABAA receptors with potency and efficacy comparable to its endogenous analog 3a,5a-P (Carter et al., 1997). As with 3a,5a-P, Ganaxolone potentiation of the GABAA receptor occurs at a site distinct from the benzodiazepine site. Ganaxolone has protective activity in diverse rodent seizure models, including clonic seizures induced by pentylenetetrazol (PTZ) and bicuculline (BIC), limbic seizures in the 6 Hz model, and amygdala kindled seizures (Carter et al., 1997; Rogawski and Reddy, 2004; Kaminski et al., 2004).


5.2 MeSH Pharmacological Classification

GABA Modulators

Substances that do not act as agonists or antagonists but do affect the GAMMA-AMINOBUTYRIC ACID receptor-ionophore complex. GABA-A receptors (RECEPTORS, GABA-A) appear to have at least three allosteric sites at which modulators act: a site at which BENZODIAZEPINES act by increasing the opening frequency of GAMMA-AMINOBUTYRIC ACID-activated chloride channels; a site at which BARBITURATES act to prolong the duration of channel opening; and a site at which some steroids may act. GENERAL ANESTHETICS probably act at least partly by potentiating GABAergic responses, but they are not included here. (See all compounds classified as GABA Modulators.)


Neurosteroids

Endogenous compounds or drugs that affect neuronal excitability through modulation of specific ionotropic receptors (e.g., GABA-A RECEPTORS). Endogenous neurosteroids are steroid hormones de novo synthesized by neurons and glial cells from steroid metabolite precursors (e.g., PREGNENOLONE). (See all compounds classified as Neurosteroids.)


5.3 ATC Code

N - Nervous system

N03 - Antiepileptics

N03A - Antiepileptics

N03AX - Other antiepileptics

N03AX27 - Ganaxolone


5.4 Biological Half-Life

1.3-1.9 hours


5.5 Mechanism of Action

Ganaxolone belongs to a novel class of neuroactive steroids called epalons, which specifically modulate the -aminobutyric acid type A (GABAA) receptor in the central nervous system (CNS). Chemically related to progesterone but devoid of any hormonal activity, the epalons have potent antiepileptic, anxiolytic, sedative, and hypnotic activities in animals.


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Nuray Chemicals Private Limited

India

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Pfizer CentreOne

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Curia

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Curia

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Sterling Spa

Italy

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ScinoPharm Taiwan Ltd

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GANAXOLONE

NDC Package Code : 0009-1005

Start Marketing Date : 2023-10-25

End Marketing Date : 2025-12-31

Dosage Form (Strength) : POWDER (1kg/kg)

Marketing Category : BULK INGREDIENT

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GANAXOLONE

NDC Package Code : 65129-3162

Start Marketing Date : 2021-06-01

End Marketing Date : 2025-12-31

Dosage Form (Strength) : POWDER (1kg/kg)

Marketing Category : BULK INGREDIENT

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Ganaxolone

About the Company : Established in 2012, Nuray Chemicals Pvt Ltd is an API manufacturer for highly regulated markets. Its manufacturing facility with state-of-the-art R&D is located near Chennai in th...

Established in 2012, Nuray Chemicals Pvt Ltd is an API manufacturer for highly regulated markets. Its manufacturing facility with state-of-the-art R&D is located near Chennai in the Indian state of Tamil Nadu. Nuray’s expertise and experience lie in developing the chemistry requirements of the pharmaceutical industry, including the synthesis of NCEs, impurities, metabolites, method development, advanced intermediates and APIs for commercial launch. With its focus on maintaining the highest levels of quality, reliability and transparency, Nuray has emerged as a trusted partner in the global generic market.
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Curia

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Ganaxolone

About the Company : Curia is a global contract research, development and manufacturing organization (CDMO), offering products and services across the drug development spectrum to help our partners tur...

Curia is a global contract research, development and manufacturing organization (CDMO), offering products and services across the drug development spectrum to help our partners turn their ideas into real-world impact. We partner closely with pharmaceutical and biotechnology companies to boost business performance and improve patients’ lives. From early discovery and development through manufacturing and commercialization, our suite of custom solutions allows us to tailor every engagement to your precise needs, whether that’s an independent project or an opportunity that cuts across the drug continuum.
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Ganaxolone

About the Company : Curia is a global contract research, development and manufacturing organization (CDMO), offering products and services across the drug development spectrum to help our partners tur...

Curia is a global contract research, development and manufacturing organization (CDMO), offering products and services across the drug development spectrum to help our partners turn their ideas into real-world impact. We partner closely with pharmaceutical and biotechnology companies to boost business performance and improve patients’ lives. From early discovery and development through manufacturing and commercialization, our suite of custom solutions allows us to tailor every engagement to your precise needs, whether that’s an independent project or an opportunity that cuts across the drug continuum.
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Sterling Spa

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Ganaxolone

About the Company : The Quality Assurance department is the Guardian of Quality within the Company. We have a team dedicated to identifying and implementing new ICH guidelines. Our team is prepared ...

The Quality Assurance department is the Guardian of Quality within the Company. We have a team dedicated to identifying and implementing new ICH guidelines. Our team is prepared to obtain quality objectives which have been universally recognized by Inspection Authorities and the most important Pharmaceutical Companies from around the globe. This system is periodically verified through internal, national and international inspectors who have been able to recognize the high level obtained by the system and the actual achievement of quality objectives multiple times. Sterling has been inspected three times by FDA .
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Drugs in Development

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Details:

under the termination agreement, Marinus regains Ztalmy (ganaxolone) rights in Europe. It is approved for the adjunctive treatment of epileptic seizures associated with CDKL5 deficiency disorder.


Lead Product(s): Ganaxolone

Therapeutic Area: Neurology Brand Name: Ztalmy

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Marinus Pharmaceuticals

Deal Size: $1.5 million Upfront Cash: Undisclosed

Deal Type: Termination December 30, 2024

Fermion Orion Company Banner

01

Details : under the termination agreement, Marinus regains Ztalmy (ganaxolone) rights in Europe. It is approved for the adjunctive treatment of epileptic seizures associated with CDKL5 deficiency disorder.

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : Undisclosed

December 30, 2024

Fermion Orion Company Banner

Details:

Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is used for the treatment of epileptic seizures associated with CDKL5 deficiency disorder.


Lead Product(s): Ganaxolone

Therapeutic Area: Rare Diseases and Disorders Brand Name: Ztalmy

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Marinus Pharmaceuticals

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable July 31, 2023

Fermion Orion Company Banner

02

Details : Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is used for the treatment of epileptic seizures associated with CDKL5 deficiency disorder.

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

July 31, 2023

Fermion Orion Company Banner

Details:

Through the acquisition, Immedica strengthens it's global rare disease business by adding Ztalmy (ganaxalone). It is indicated for the treatment of seizures associated with CDKL5 deficiency disorder.


Lead Product(s): Ganaxolone

Therapeutic Area: Neurology Brand Name: Ztalmy

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Immedica Pharma

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Acquisition December 30, 2024

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American Thoracic Society
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American Thoracic Society
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Details : Through the acquisition, Immedica strengthens it's global rare disease business by adding Ztalmy (ganaxalone). It is indicated for the treatment of seizures associated with CDKL5 deficiency disorder.

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : Undisclosed

December 30, 2024

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Details:

Ztalmy (ganaxolone) is a neuroactive steroid that targets GABAA receptors via a unique binding site. It is being evaluated for the treatment of TSC-related epilepsy.


Lead Product(s): Ganaxolone

Therapeutic Area: Neurology Brand Name: Ztalmy

Study Phase: Phase IIIProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable October 24, 2024

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04

American Thoracic Society
Not Confirmed
American Thoracic Society
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Details : Ztalmy (ganaxolone) is a neuroactive steroid that targets GABAA receptors via a unique binding site. It is being evaluated for the treatment of TSC-related epilepsy.

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

October 24, 2024

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Details:

Ztalmy (ganaxolone) is a neuroactive steroid GABAA receptor PAM, indicated for epileptic seizures in patients two years of age and older with CDKL5 deficiency disorder.


Lead Product(s): Ganaxolone

Therapeutic Area: Neurology Brand Name: Ztalmy

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Tenacia Biotechnology

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable July 18, 2024

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05

American Thoracic Society
Not Confirmed
American Thoracic Society
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Details : Ztalmy (ganaxolone) is a neuroactive steroid GABAA receptor PAM, indicated for epileptic seizures in patients two years of age and older with CDKL5 deficiency disorder.

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

July 18, 2024

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Details:

Ztalmy (ganaxolone) is a neuroactive steroid that targets GABAA receptors via a unique binding site. It is being evaluated for the treatment of refractory status epilepticus.


Lead Product(s): Ganaxolone

Therapeutic Area: Neurology Brand Name: Ztalmy

Study Phase: Phase IIIProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable June 17, 2024

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06

American Thoracic Society
Not Confirmed
American Thoracic Society
Not Confirmed

Details : Ztalmy (ganaxolone) is a neuroactive steroid that targets GABAA receptors via a unique binding site. It is being evaluated for the treatment of refractory status epilepticus.

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

June 17, 2024

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Details:

Ztalmy (ganaxolone) is a neuroactive steroid modulating both synaptic and extrasynaptic GABAA receptors to treat refractory status epilepticus.


Lead Product(s): Ganaxolone

Therapeutic Area: Neurology Brand Name: Ztalmy

Study Phase: Phase IIIProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable April 15, 2024

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07

American Thoracic Society
Not Confirmed
American Thoracic Society
Not Confirmed

Details : Ztalmy (ganaxolone) is a neuroactive steroid modulating both synaptic and extrasynaptic GABAA receptors to treat refractory status epilepticus.

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

April 15, 2024

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Details:

Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is investigated for the treatment of Seizures Associated With CDKL5 Deficiency Disorder.


Lead Product(s): Ganaxolone

Therapeutic Area: Rare Diseases and Disorders Brand Name: Ztalmy

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable May 26, 2023

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08

American Thoracic Society
Not Confirmed
American Thoracic Society
Not Confirmed

Details : Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is investigated for the treatment of Seizures Associated With CDKL5 Deficiency Disorder.

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

May 26, 2023

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Details:

Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is investigated for the treatment of lennox-gastaut syndrome.


Lead Product(s): Ganaxolone

Therapeutic Area: Rare Diseases and Disorders Brand Name: Ztalmy

Study Phase: PreclinicalProduct Type: Other Small Molecule

Sponsor: Inapplicable

Deal Size: Inapplicable Upfront Cash: Inapplicable

Deal Type: Inapplicable March 22, 2023

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09

American Thoracic Society
Not Confirmed
American Thoracic Society
Not Confirmed

Details : Ztalmy (ganaxolone) is a positive allosteric modulation of the gamma-aminobutyric acid type A (GABAA) receptor in the CNS. It is investigated for the treatment of lennox-gastaut syndrome.

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : Inapplicable

March 22, 2023

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Details:

Under the terms of the agreement, Tenacia will receive exclusive rights to develop and commercialize certain formulations of Ztalmy (ganaxolone) in Mainland China, Hong Kong, Macau and Taiwan, including the current oral and intravenous (IV) dose formulations.


Lead Product(s): Ganaxolone

Therapeutic Area: Neurology Brand Name: Ztalmy

Study Phase: Approved FDFProduct Type: Other Small Molecule

Sponsor: Tenacia Biotechnology

Deal Size: $266.0 million Upfront Cash: $10.0 million

Deal Type: Collaboration November 17, 2022

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10

American Thoracic Society
Not Confirmed
American Thoracic Society
Not Confirmed

Details : Under the terms of the agreement, Tenacia will receive exclusive rights to develop and commercialize certain formulations of Ztalmy (ganaxolone) in Mainland China, Hong Kong, Macau and Taiwan, including the current oral and intravenous (IV) dose formulat...

Product Name : Ztalmy

Product Type : Other Small Molecule

Upfront Cash : $10.0 million

November 17, 2022

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FDF Dossiers

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American Thoracic Society
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GANAXOLONE

Brand Name : ZTALMY

Dosage Form : SUSPENSION;ORAL

Dosage Strength : 50MG/ML

Packaging :

Approval Date : 2022-06-01

Application Number : 215904

Regulatory Info : RX

Registration Country : USA

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FDA Orange Book

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MARINUS

U.S.A
American Thoracic Society
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MARINUS

U.S.A
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American Thoracic Society
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GANAXOLONE

Brand Name : ZTALMY

Dosage Form : SUSPENSION;ORAL

Dosage Strength : 50MG/ML

Approval Date : 2022-06-01

Application Number : 215904

RX/OTC/DISCN : RX

RLD : Yes

TE Code :

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FDA approvals rise 49% in 2023; CRISPR’s gene editing therapy sees light of day
In 2022, when the US Food and Drug Administration (FDA) was reeling under the impact of the pandemic, new drug approvals by the agency dropped by 26 percent. But last year, FDA’s new drug approvals rebounded by an impressive 49 percent, with the Center for Drug Evaluation and Research (CDER) approving 55 new drugs in 2023. Of them, 36 percent were considered first-in-class, while small molecules made up for 62 percent of the total drugs approved (i.e. 34). FDA’s Center for Biologics Evaluation and Research (CBER) okayed 19 biologics in 2023 compared to eight in the previous year.The first half of 2023 saw the debut of vaccines for the all-too-common respiratory syncytial virus (RSV). Among the other notable approvals in H1 was Biogen and Eisai’s Alzheimer’s drug Leqembi (lecanemab). Out of the total 55 drug approvals, 29 came in H2 2023. This includes Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy that relies on the Nobel Prize-winning CRISPR gene-editing technology. Casgevy has been approved as a treatment for sickle-cell disease (SCD) and β-thalassemia.While FDA witnessed a sharp rise in approvals in 2023, many other drug regulators didn’t. The European Medicines Agency (EMA) granted marketing authorization to 32 novel drugs in 2023, a fall from 33 in 2022. Similarly, Health Canada’s approvals in 2023 decreased to 38, compared to 45 in the previous year.As usual, oncology topped the list of drug approvals by therapeutic area, at 39 (as opposed to 35 in 2022). Rare diseases was the second most popular therapeutic area for drug approvals. With drugmakers clearly paying heed to the unmet needs of patients suffering from rare diseases, this therapeutic area sprinted from a 9 percent share and the fourth position among new approvals in 2022 to an impressive 34 percent share in 2023. A quarter of the new drug approvals were in infectious diseases, followed by immunology (19 percent) and neurology (7 percent).View New Drug Approvals in 2023 with Estimated Sales (Free Excel Available) Casgevy, postpartum depression drug Zurzuvae emerge as potential blockbustersGene therapy Casgevy, postpartum depression (PPD) med Zurzuvae, blood cancer med Elrexfio and ulcerative colitis drug Velsipity were some of the prominent approvals of 2023.Britain’s Medicines and Healthcare products Regulatory Agency was the first to okay Casgevy in November as a cure for SCD and β-thalassemia. Soon, the FDA approved it for SCD. In January this year, the American agency also approved it for transfusion-dependent β-thalassemia (TDT). Analysts estimate Casgevy to generate US$ 2.6 billion in peak sales, says Nature. Biogen and Sage’s PPD therapy Zurzuvae became the first and only FDA-approved pill for the condition that can be life-threatening for both the mother and the child. Global sales of Zurzuvae are forecast to hit US$ 1.28 billion by 2028.In August, Pfizer’s Elrexfio (elranatamab) became the first “off-the-shelf” (ready-to-use) therapy in the US for multiple myeloma. The drug provides an option for patients with hard to treat or relapsed blood cancer and is estimated to bring in US$ 861 million in peak sales by 2028, says Nature.Pfizer also bagged another significant approval in October — its drug Velsipity (etrasimod) was greenlit by the FDA to treat adults with ulcerative colitis, an inflammatory bowel disease. Peak revenue for Velsipity is expected to come in at US$ 825 million, as per Evaluate.View New Drug Approvals in 2023 with Estimated Sales (Free Excel Available) Astra’s Truqap, GSK’s Ojjaara among top cancer therapies given FDA nod in H2In November, FDA approved AstraZeneca’s Truqap (capivasertib) in combination with the Anglo-Swedish drugmaker’s Faslodex (fulvestrant) for treating adult patients with hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer with one or more biomarker alterations. Evaluate Pharma forecasts peak Truqap sales to come in at about US$ 690 million.In September, FDA approved GSK’s Ojjaara (momelotinib) as the first and only treatment for myelofibrosis with anemia. Nearly all myelofibrosis patients are estimated to develop anemia over the course of the disease. Ojjaara is taken orally once a day.Other notable oncology treatments okayed by FDA in H2 2023 include Daiichi’s Vanflyta (quizartinib) in July to treat an aggressive blood cancer known as acute myeloid leukemia (AML). In August, FDA approved Janssen’s bispecific antibody Talvey (talquetamab-tgvs) for difficult-to-treat blood cancer. The agency approved two cancer therapies in November — BMS’ Augtyro (repotrectinib) for ROS1-positive non-small cell lung cancer (NSCLC) and Takeda’s targeted oral therapy Fruzaqla (fruquintinib) for adult patients with metastatic colorectal cancer (mCRC).View New Drug Approvals in 2023 with Estimated Sales (Free Excel Available) Rare disease drugs Santhera-Catalyst’s Agamree, Novo’s Rivfloza bag approval in H2In October, FDA approved Santhera Pharmaceuticals and Catalyst Pharma’s Agamree (vamorolone), an oral suspension treatment for Duchenne muscular dystrophy (DMD) in patients two years of age and older. This makes it the first drug fully approved in both the US and Europe for the muscle degeneration disorder. Agamree acts in a manner similar to other steroids, which are the standard of care for the inherited rare disease. However, it causes fewer side effects.FDA also okayed Novo Nordisk’s once-a-month injection Rivfloza (nedosiran) in October to treat a rare genetic condition — primary hyperoxaluria type 1 (PH1) — that causes recurring kidney stones.In November, the agency approved Takeda’s Adzynma (ADAMTS13, recombinant-krhn) as the first treatment for both adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), a rare genetic blood disorder. Other noteworthy FDA approvals in H2 2023 for rare blood diseases include Novartis’ Fabhalta and bluebird bio's Lyfgenia. Fabhalta is the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria, a rare disease that causes symptoms such as hemolytic anemia, hemoglobinuria (excretion of hemoglobin in the urine), fatigue, shortness of breath etc. Lyfgenia is the first cell-based gene therapy for the treatment of SCD in patients 12 years and older. Similarly, another rare disease drug — Regeneron’s Veopoz —  bagged FDA approval in August last. Veopoz treats CHAPLE disease, an ultra-rare disease in which patients have severe gastrointestinal problems.View New Drug Approvals in 2023 with Estimated Sales (Free Excel Available) Our viewAfter a lull in 2022, new drug approvals have finally gathered momentum. The good news is that this year, several pathbreaking drugs are coming up for approval, such as Madrigal Pharmaceuticals’ resmetirom (the first treatment for NASH with liver fibrosis), Merck’s sotatercept (a treatment for pulmonary arterial hypertension), Lilly’s donanemab for Alzheimer’s disease and Karuna Therapeutics’ drug to treat schizophrenia. Let’s hope 2024 turns out to be an even bigger year for new drug approvals.

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Looking for 38398-32-2 / Ganaxolone API manufacturers, exporters & distributors?

Ganaxolone manufacturers, exporters & distributors 1

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PharmaCompass offers a list of Ganaxolone API manufacturers, exporters & distributors, which can be sorted by GMP, USDMF, JDMF, KDMF, CEP (COS), WC, Price,and more, enabling you to easily find the right Ganaxolone manufacturer or Ganaxolone supplier for your needs.

Send us enquiries for free, and we will assist you in establishing a direct connection with your preferred Ganaxolone manufacturer or Ganaxolone supplier.

PharmaCompass also assists you with knowing the Ganaxolone API Price utilized in the formulation of products. Ganaxolone API Price is not always fixed or binding as the Ganaxolone Price is obtained through a variety of data sources. The Ganaxolone Price can also vary due to multiple factors, including market conditions, regulatory modifications, or negotiated pricing deals.

API | Excipient name

Ganaxolone

Synonyms

38398-32-2, Ccd 1042, Ztalmy, Ccd-1042, 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one, 98wi44ohiq

Cas Number

38398-32-2

Unique Ingredient Identifier (UNII)

98WI44OHIQ

About Ganaxolone

Ganaxolone is an orally bioavailable synthetic analog of the neuroactive steroid allopregnanolone and positive allosteric modulator of the gamma-aminobutyric acid (GABA)-A receptor, with anxiolytic, sedative, antidepressant and anticonvulsant activities. Upon oral administration, ganaxolone specifically targets and binds to the allosteric sites of the synaptic and extrasynaptic GABA-A receptor-chloride ionophore complex in the central nervous system (CNS). This leads to an increase in the opening of chloride channels, membrane hyperpolarization, increases the inhibitory effect of GABA on the CNS, and inhibits neurotransmission. This blocks seizure propagation and elevates seizure thresholds.

Ganaxolone Manufacturers

A Ganaxolone manufacturer is defined as any person or entity involved in the manufacture, preparation, processing, compounding or propagation of Ganaxolone, including repackagers and relabelers. The FDA regulates Ganaxolone manufacturers to ensure that their products comply with relevant laws and regulations and are safe and effective to use. Ganaxolone API Manufacturers are required to adhere to Good Manufacturing Practices (GMP) to ensure that their products are consistently manufactured to meet established quality criteria.

click here to find a list of Ganaxolone manufacturers with USDMF, JDMF, KDMF, CEP, GMP, COA and API Price related information on PhamaCompass.

Ganaxolone Suppliers

A Ganaxolone supplier is an individual or a company that provides Ganaxolone active pharmaceutical ingredient (API) or Ganaxolone finished formulations upon request. The Ganaxolone suppliers may include Ganaxolone API manufacturers, exporters, distributors and traders.

click here to find a list of Ganaxolone suppliers with USDMF, JDMF, KDMF, CEP, GMP, COA and API Price related information on PharmaCompass.

Ganaxolone NDC

National Drug Code is a comprehensive database maintained by the FDA that contains information on all drugs marketed in the US. This directory includes information about finished drug products, unfinished drug products, and compounded drug products, including those containing Ganaxolone as an active pharmaceutical ingredient (API).

Finished drug products

The FDA updates the NDC directory daily. The NDC numbers for Ganaxolone API and other APIs are published in this directory by the FDA.

Unfinished drugs

The NDC unfinished drugs database includes product listing information submitted for all unfinished drugs, such as active pharmaceutical ingredients (APIs), drugs intended for further processing and bulk drug substances for compounding.

Pharmaceutical companies that manufacture Ganaxolone as an active pharmaceutical ingredient (API) must furnish the FDA with an updated record of all drugs that they produce, prepare, propagate, compound, or process for commercial distribution in the US at their facilities.

Compounded drug products

The NDC directory also contains data on finished compounded human drug products that contain Ganaxolone and are produced by outsourcing facilities. While these outsourcing facilities are not mandated to assign a Ganaxolone NDC to their finished compounded human drug products, they may choose to do so.

click here to find a list of Ganaxolone suppliers with NDC on PharmaCompass.

Ganaxolone GMP

Ganaxolone Active pharmaceutical ingredient (API) is produced in GMP-certified manufacturing facility.

GMP stands for Good Manufacturing Practices, which is a system used in the pharmaceutical industry to make sure that goods are regularly produced and monitored in accordance with quality standards. The FDA’s current Good Manufacturing Practices requirements are referred to as cGMP or current GMP which indicates that the company follows the most recent GMP specifications. The World Health Organization (WHO) has its own set of GMP guidelines, called the WHO GMP. Different countries can also set their own guidelines for GMP like China (Chinese GMP) or the EU (EU GMP).

PharmaCompass offers a list of Ganaxolone GMP manufacturers, exporters & distributors, which can be sorted by USDMF, JDMF, KDMF, CEP (COS), WC, API price, and more, enabling you to easily find the right Ganaxolone GMP manufacturer or Ganaxolone GMP API supplier for your needs.

Ganaxolone CoA

A Ganaxolone CoA (Certificate of Analysis) is a formal document that attests to Ganaxolone's compliance with Ganaxolone specifications and serves as a tool for batch-level quality control.

Ganaxolone CoA mostly includes findings from lab analyses of a specific batch. For each Ganaxolone CoA document that a company creates, the USFDA specifies specific requirements, such as supplier information, material identification, transportation data, evidence of conformity and signature data.

Ganaxolone may be tested according to a variety of international standards, such as European Pharmacopoeia (Ganaxolone EP), Ganaxolone JP (Japanese Pharmacopeia) and the US Pharmacopoeia (Ganaxolone USP).

Inform the supplier about your product requirements, specifying if you need a product with particular monograph like EP (Ph. Eur.), USP, JP, BP, or any other quality. In addition, clarify whether you need hydrochloride (HCl), anhydricum, base, micronisatum or a specific level of purity. To find reputable suppliers, utilize the filters and select those certified by GMP, FDA, or any other certification as per your requirement.
For your convenience, we have listed synonyms and CAS numbers to help you find the best supplier. The use of synonyms and CAS numbers can be helpful in identifying potential suppliers, but it is crucial to note that they might not always indicate the exact same product. It is important to confirm the product details with the supplier before making a purchase to ensure that it meets your requirements.
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