Ataluren

PTC Therapeutics announced FDA's acceptance of the Translarna™ NDA resubmission for treating nonsense mutation Duchenne muscular dystrophy. The submission is based on significant benefits shown in Study 041 and the STRIDE registry, including delayed loss of ambulation and improved lung function. Translarna targets the underlying cause of nmDMD, offering hope to affected families.

DelveInsight's 'Duchenne Muscular Dystrophy (DMD) Pipeline Insight, 2024' analyzes over 75 pipeline drugs from 75+ companies, focusing on recent FDA, EMA, and PMDA approvals, clinical trials, emerging therapies, and key players like Roche, Santhera, and Sarepta. The report covers various stages of development, routes of administration, and mechanisms of action, offering insights into the evolving DMD therapeutics landscape.

The European Medicine Agency's CHMP confirmed its recommendation to not renew Translarna's conditional marketing authorisation, concluding its effectiveness for treating Duchenne muscular dystrophy caused by nonsense mutations remains unconfirmed.

The CHMP maintained its recommendation not to renew Translarna's conditional marketing authorization for treating nonsense mutation Duchenne muscular dystrophy, despite PTC Therapeutics' commitment to keep it available. The European Commission is expected to make a final decision within two months.

Catumaxomab, now branded as Korjuny, received a positive opinion from the EMA's CHMP for treating malignant ascites, marking its return after a decade off the market. Korjuny, a bispecific and trifunctional antibody, engages Epcam on cancer cells and CD3 on T cells, recruiting immune cells to destroy tumor cells. The EMA approval is limited to malignant ascites, but ongoing trials aim to expand its use, including a global phase III trial in advanced gastric cancer.

EMA won't renew Translarna's permit; Novartis pays $70M for a cancer drug; Amylyx's AMX0035 shows promise in rare disease; FDA delays Ocaliva approval; Australia rejects Leqembi; Amicus settles with Teva over Galafold.

The committee recommended marketing authorisations for ten new medicines, including treatments for haemophilia, influenza, malignant ascites, tuberculosis diagnosis, hereditary transthyretin-mediated amyloidosis, and opioid dependence. Six existing medicines received extensions of indication, while two applications were withdrawn. The CHMP confirmed refusals for conditional marketing authorisations for masitinib and non-renewal for Translarna. Updates to COVID-19 vaccines were also approved. Outi Mäki-Ikola was elected as the new CHMP vice-chair.

EMA's CHMP confirms non-renewal of Translarna's conditional marketing authorisation due to unconfirmed effectiveness in treating Duchenne muscular dystrophy caused by nonsense mutations.