Drugs in Dev. Genetic Disease
Phase IV
United States 
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
New Biomarker Data Support Potential Benefit of SPINRAZA in Infants
Details : Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
Product Name : Spinraza
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
June 03, 2024
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
CHMP Recommends Roche’s Evrysdi for Babies Under Two Months Old with Spinal Muscular Atrophy (SMA)
Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.
Product Name : Evrysdi
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
July 21, 2023
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Evrysdi (risdiplam) is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency.
Product Name : Evrysdi
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
June 30, 2023
Lead Product(s) : Risdiplam
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Lanadelumab
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The observational Phase 4 EMPOWER study, evaluating real-world HAE attack rates before and after treatment with TAKHZYRO in patients with HAE types I and II, is ongoing. Full results of the EMPOWER study are expected to be published in 2024.
Product Name : Takhzyro
Product Type : Antibody
Upfront Cash : Inapplicable
February 28, 2022
Lead Product(s) : Lanadelumab
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The RESPOND study aims to evaluate if Spinraza can benefit SMA patients who have experienced a ‘suboptimal’ response to Zolgensma (onasemnogene abeparvovec). It will be conducted at around 20 sites globally, with researchers aiming to enrol up to 60 ...
Product Name : Spinraza
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
August 01, 2021
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Recombinant Coagulation Factor Ix
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Medexus reached the 50% enrollment target in its Phase 4 clinical trial investigating IXINITY® as a prophylactic treatment for pediatric patients under 12 years of age with hemophilia B.
Product Name : Ixinity
Product Type : Protein
Upfront Cash : Inapplicable
November 16, 2020
Lead Product(s) : Recombinant Coagulation Factor Ix
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The RESPOND study will seek to understand if the proven efficacy of SPINRAZA and its continuous production of SMN protein may also benefit patients previously treated with gene therapy.
Product Name : Spinraza
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
July 21, 2020
Lead Product(s) : Nusinersen Sodium
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The results of this study demonstrate that the triple combination provides significant additional benefit compared to existing CFTR modulator therapy for F/G and F/RF patients and supports the benefit of the medicine for patients with at least one F508de...
Product Name : Kaftrio
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
July 20, 2020
Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor
Therapeutic Area : Genetic Disease
Study Phase : Phase IV
Sponsor : Vertex Pharmaceuticals
Deal Size : Undisclosed
Deal Type : Agreement
Vertex Announces Expansion of Reimbursement Agreement With NHS England
Details : As part of the agreement with NHS England, Vertex has committed to submit ORKAMBI®, SYMKEVI® and KAFTRIO® to the National Institute for Health and Care Excellence (NICE) within an agreed upon timetable, allowing for a period of real-world data collect...
Product Name : Kaftrio
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
June 30, 2020
Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor
Therapeutic Area : Genetic Disease
Highest Development Status : Phase IV
Sponsor : Vertex Pharmaceuticals
Deal Size : Undisclosed
Deal Type : Agreement
Reset All
