Ataluren

Generic Name: Ataluren

Brand Names: Translarna

Drug Type: Small Molecule

Chemical Formula: C_₁₅H_₉FN_₂O_₃

CAS Number: 775304-57-9

Unique Ingredient Identifier: K16AME9I3V

Background: Ataluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.

This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.

Indication: Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.

Associated Conditions: Duchenne Muscular Dystrophy (DMD)

Associated Therapies: -

Clinical Trials

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

EMBARK trial (SRP-9001-301) assessed delandistrogene moxeparvovec safety/efficacy in DMD patients aged 4-7. Conducted at 42 sites, it was a phase 3, two-part, multinational, randomized, double-blind, placebo-controlled trial. Patients received either delandistrogene moxeparvovec or placebo, with crossover in Part 2. Primary endpoint was change in NSAA total score from baseline to week 52. Key secondary endpoints included micro-dystrophin expression at week 12, and changes in TTR and 10MWR from baseline to week 52.

Related Clinical Trials:

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

investing.com

3 months ago

FDA accelerates PTC Therapeutics Huntington's treatment

PTC Therapeutics' PTC518 for Huntington's disease granted Fast Track designation by FDA; interim data from PIVOT-HD study shows 43% reduction in mutant Huntingtin protein levels at 10-mg dose, with favorable clinical effects and good tolerability. PTC Therapeutics reports $187 million in Q2 2024 revenue, updates full-year guidance to $700-$750 million, and makes progress in clinical trials and regulatory submissions. Stock performance shows 62.12% return over the last year, but caution advised due to overbought RSI.

ema.europa.eu

6 months ago

EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy treatment

EMA’s CHMP recommended not renewing Translarna’s EU marketing authorisation for Duchenne muscular dystrophy, citing insufficient evidence of effectiveness despite reviewing additional data and acknowledging the high unmet medical need.

benzinga.com

7 months ago

Pfizer Phase III DMD Gene Therapy Study Misses Goal

Pfizer's phase III study on fordadistrogene movaparvovec for Duchenne muscular dystrophy (DMD) failed primary and key secondary endpoints. The therapy aimed to improve motor function in boys aged 4-8. Despite safety concerns, Pfizer plans to share detailed results soon. Sarepta Therapeutics leads the DMD treatment market.

nasdaq.com

9 months ago

PTC Therapeutics Announces FDA Acceptance of Translarna™ NDA Resubmission for Nonsense Mutation Duchenne Muscular Dystrophy

PTC Therapeutics announced FDA's acceptance of the NDA resubmission for Translarna™, targeting nonsense mutation Duchenne muscular dystrophy. The submission is based on significant benefits shown in Study 041 and long-term treatment benefits in the STRIDE registry, highlighting a delay in loss of ambulation.

parentprojectmd.org

10 months ago

PTC Therapeutics Announces Plans to Resubmit NDA for Translarna™ by Mid-2024

PTC Therapeutics plans to resubmit an NDA for Translarna™ by mid-2024, following FDA feedback. The therapy targets nonsense mutation Duchenne. PPMD supports FDA's review for informed patient decisions, hopeful for approval.