United States

Vertex Pharmaceuticals

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

Vertex Announces Positive Phase 3 Study for TRIKAFTA® in Children Ages 6-11 Years With Cystic Fibrosis

Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

Therapeutic Area: Genetic Disease Product Name: Trikafta

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 10, 2020

Details:

The primary endpoint of the study was safety and tolerability, and the results showed that TRIKAFTA was generally well tolerated and the safety data were consistent with those observed in previous Phase 3 studies.

Option Care Health

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

Option Care Health Selected as a Limited Distribution Provider of VILTEPSO™ Injection for Patients with Duchenne Muscular Dystrophy

Lead Product(s): Viltolarsen

Therapeutic Area: Genetic Disease Product Name: Viltepso

Highest Development Status: Approved Product Type: Large molecule

Partner/Sponsor/Collaborator: NS PHARMA INC

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Agreement September 02, 2020

Details:

Option Care Health has been selected to participate in the limited distribution network of VILTEPSO™ (viltolarsen) for patients with Duchenne Muscular Dystrophy (“DMD”) who are amenable to exon 53 skipping therapy.

Vertex Pharmaceuticals

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

FDA Accepts Vertex’s Supplemental New Drug Applications for TRIKAFTA® , SYMDEKO® and KALYDECO® for Additional CFTR Mutations

Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

Therapeutic Area: Genetic Disease Product Name: Trikafta

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 01, 2020

Details:

U.S. Food and Drug Administration (FDA) accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor).

Recordati Rare Diseases

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

U.S. FDA Approves CYSTADROPS® 0.37%, A New Practical Treatment Option for the Ocular Manifestations of Cystinosis

Lead Product(s): Cysteamine Hydrochloride

Therapeutic Area: Genetic Disease Product Name: Cystadrops

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable August 25, 2020

Details:

CYSTADROPS demonstrated a significant reduction in cystine crystal deposits in the cornea of the eye and is the first and only FDA-approved cysteamine drop formulation with four times a day dosing.

Vertex Pharmaceuticals

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

Vertex’s Kaftrio Approved for Use in Europe and Made Accessible on the NHS

Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor

Therapeutic Area: Genetic Disease Product Name: Kaftrio

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable August 21, 2020

Details:

E.C has granted marketing authorization for KAFTRIO® in a combination regimen with ivacaftor to treat people with cystic fibrosis ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene.

Emmaus Life Sciences

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

Emmaus Life Sciences Announces Submission of Marketing Authorization Application to the Saudi Food and Drug Authority

Lead Product(s): L-Glutamine

Therapeutic Area: Genetic Disease Product Name: Endari

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable August 18, 2020

Details:

The application follows Emmaus’ announcement that the SFDA had granted Endari® a priority review designation as part of its program to expedite the review of drugs that are expected to have a significant impact on the treatment of a disease with unmet medical need.

NS PHARMA INC

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

NS Pharma's VILTEPSO injection Now FDA-Approved in the U.S. for the Treatment of Duchenne Muscular Dystrophy

Lead Product(s): Viltolarsen

Therapeutic Area: Genetic Disease Product Name: Viltepso

Highest Development Status: Approved Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable August 12, 2020

Details:

U.S. Food & Drug Administration (FDA) has approved VILTEPSO™ (viltolarsen) injection for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy.

Emmaus Life Sciences

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

Emmaus Life Sciences Receives Scientific Advice From the EMA Regarding Xyndari™

Lead Product(s): L-Glutamine

Therapeutic Area: Genetic Disease Product Name: Xyndari

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 29, 2020

Details:

European Medicines Agency (EMA) issued scientific advice to Emmaus regarding the clinical development pathway for Xyndari™ for the treatment of sickle cell disease.

Genentech

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

New Data From Phase IIIb Study Reinforces Safety Profile of Genentech’s Hemlibra (emicizumab-kxwh) in People With Hemophilia A

Lead Product(s): Emicizumab

Therapeutic Area: Genetic Disease Product Name: Hemlibra

Highest Development Status: Approved Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 12, 2020

Details:

In the STASEY study, Hemlibra was effective with no new safety signals identified in adults and adolescents with hemophilia A with factor VIII inhibitors, which was consistent with previous safety observations.

Orsini Pharmaceutical

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

World Dairy Expo

Participation Not Confirmed

Update your events

Orsini Pharmaceutical Services Expands Partnership With Ultragenyx To Provide Dojolvi™ (triheptanoin)

Lead Product(s): Triheptanoin

Therapeutic Area: Genetic Disease Product Name: Dojolvi

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Partnership July 10, 2020

Details:

Orsini Pharmaceutical Services has been selected by Ultragenyx Pharmaceutical as a limited distribution specialty pharmacy partner for Dojolvi (triheptanoin). Dojolvi™ is indicated as a source of calories and fatty acids for the treatment of molecularly confirmed LC-FAOD.

Find

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

Product Type

Companies

Vertex Pharmaceuticals

Vertex Announces Positive Phase 3 Study for TRIKAFTA® in Children Ages 6-11 Years With Cystic Fibrosis

Option Care Health

Option Care Health Selected as a Limited Distribution Provider of VILTEPSO™ Injection for Patients with Duchenne Muscular Dystrophy

Vertex Pharmaceuticals

FDA Accepts Vertex’s Supplemental New Drug Applications for TRIKAFTA® , SYMDEKO® and KALYDECO® for Additional CFTR Mutations

Recordati Rare Diseases

U.S. FDA Approves CYSTADROPS® 0.37%, A New Practical Treatment Option for the Ocular Manifestations of Cystinosis

Vertex Pharmaceuticals

Vertex’s Kaftrio Approved for Use in Europe and Made Accessible on the NHS

Emmaus Life Sciences

Emmaus Life Sciences Announces Submission of Marketing Authorization Application to the Saudi Food and Drug Authority

NS PHARMA INC

NS Pharma's VILTEPSO injection Now FDA-Approved in the U.S. for the Treatment of Duchenne Muscular Dystrophy

Emmaus Life Sciences

Emmaus Life Sciences Receives Scientific Advice From the EMA Regarding Xyndari™

Genentech

New Data From Phase IIIb Study Reinforces Safety Profile of Genentech’s Hemlibra (emicizumab-kxwh) in People With Hemophilia A

Orsini Pharmaceutical

Orsini Pharmaceutical Services Expands Partnership With Ultragenyx To Provide Dojolvi™ (triheptanoin)