MHRA approves Santhera’s Duchenne muscular dystrophy drug Agamree

2024-01-16
PMLiVE
上市批准临床结果引进/卖出
MHRA approves Santhera’s Duchenne muscular dystrophy drug Agamree
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来源: PMLiVE
Estimated to affect one in every 3,500 male births worldwide, DMD is a rare muscle-wasting disorder caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles.
Major milestones of the disease are the loss of ambulation and self-feeding, the start of assisted ventilation and the development of cardiomyopathy.
Agamree is a dissociative steroid that works in a similar way to existing corticosteroids, the current standard of care for children and adolescent patients with DMD, but without the same safety concerns.
The MHRA's decision on the drug, which applies to DMD patients regardless of their underlying mutation or ambulatory status, was supported by results from the pivotal VISION-DMD study and three open-label studies.
In VISION-DMD, patients treated with Agamree on average maintained growth similar to those treated with placebo, while those treated with the corticosteroid prednisone on average experienced growth stunting. Additionally, those who switched from prednisone to Agamree after 24 weeks were, on average, able to resume growing in height over the remainder of the study.
Unlike corticosteroids, Agamree did not result in a reduction of bone metabolism or a significant reduction in bone mineralisation in the spine after 48 weeks in the clinical studies, Santhera said.
The UK approval comes less than one month after the European Commission approved Agamree to treat DMD in patients aged four years and older. The drug was also approved by the US Food and Drug Administration at the end of October last year for use in patients aged two years and older.
Shabir Hasham, Santhera’s chief medical officer, said: “We are delighted to have secured a third approval for Agamree to treat Duchenne from a major regulatory agency… within a couple months.”
Hasham added that Santhera was “working towards” making the drug available to patients in the UK in the second half of 2024, after the National Institute for Health and Care Excellence completes its pricing review.
An exclusive licence from ReveraGen for all indications worldwide to Agamree is held by Santhera, which has out-licensed rights to the drug for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics.
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