MHRA extends access to Santhera’s DMD drug via the EAMS
The scientific opinion has been extended for one year while the drug is under regulatory review
Read More
Tag: DMD
Roche buys rights to Sarepta’s DMD gene therapy
Sarepta will receive $1.15 billion upfront and an equity investment
Read More
Pfizer ends two Duchenne muscular dystrophy drug trials
Pfizer is shutting down two clinical studies assessing the anti-myostatin monoclonal antibody domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD).
Read More
Summit shares plummet on DMD drug failure
Shares in Oxford University spin out Summit Therapeutics have taken a nosedive after the firm’s flagship drug ezutromid failed to hit targets in a mid-stage trial in patients with Duchenne muscular dystrophy (DMD).
Read More
CHMP takes negative view on Sarepta’s DMD therapy
Sarepta has indicated a delay to getting its Duchenne muscular dystrophy therapy Exondys approved in Europe, revealing that the European Medicine Agency’s Committee for Medicinal Products for Human Use’s trend vote was negative.
Read More
Santhera’s Raxone fails in MS trial
Santhera has been hit with more bad news regarding its flagship mitochondrial disease therapy idebenone, after it failed to hit targets in a trial assessing its potential in multiple sclerosis.
Read More
EU advisors reject Santhera’s Raxone for muscular dystrophy
Santhera’s attempt to win approval for Raxone as a treatment for Duchenne Muscular Dystrophy (DMD) in Europe has hit a giant setback after receiving a negative opinion from European Medicines Agency advisors.
Read More
FDA rejects PTC’s DMD drug Translarna
PTC Therapeutics will need to carry out at least one further trial to demonstrate the effectiveness of its Duchenne Muscular Dystrophy therapy Translarna if it is to have a chance of bagging its approval in the US.
Read More
DMD patients in the UK win early access to Santhera’s Raxone
Santhera Pharmaceuticals’ Raxone has become the first drug for Duchenne Muscular Dystrophy (DMD) to join the UK’s Early Access to Medicines Scheme (EAMS).
Read More
FDA nod for Duchenne drug
US regulators have issued a green light for Marathon Pharmaceuticals’ Emflaza to treat patients age five years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder affecting around 15,000 people in the country that causes progressive muscle deterioration and weakness.
Read More
FDA rejects PTC’s appeal over failed Translarna filing
PTC Therapeutics has been denied its first appeal contesting US regulators’ refusal to accept its marketing application for Translarna as a treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
Read More
FDA OKs first Duchenne muscular dystrophy drug
The US Food and Drug Administration has granted a conditional approval to Sarepta’s Exondys 51, the first drug cleared to treat patients with Duchenne muscular dystrophy (DMD).
Read More
- Liverpool and McMaster University announce £300,000 strategic research partnership
- Study finds link between malaria parasites developing antimalarial drug resistance
- Researchers develop ‘digital twin’ heart models to better monitor PAH NHS patients
- Closed Loop and Pharmanovia investigate precision medicine combination in hypertension
- US study reveals interferon combinations cause lupus symptoms and affect treatment
