Santhera’s Duchenne muscular dystrophy drug shows promise as long-term treatment

2024-02-19
PMLiVE
临床结果上市批准临床3期
Santhera’s Duchenne muscular dystrophy drug shows promise as long-term treatment
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来源: PMLiVE
Santhera Pharmaceuticals has announced the publication of results in the journal Neurology that support the long-term efficacy and safety profile of its Duchenne muscular dystrophy (DMD) drug, Agamree (vamorolone).
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, the loss of 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 adolescents with DMD, but without the same safety concerns.
The drug was approved by the Medicines and Healthcare products Regulatory Agency (MHRA) at the beginning of this year to treat DMD patients aged four years and older and has also recently been approved for varying age populations in the EU and US.
The authorisations were supported by positive results from the pivotal VISION-DMD study, in which Agamree met the primary endpoint, time to stand velocity versus placebo, after 24 weeks of treatment and showed a good safety and tolerability profile.
The latest publication in Neurology reports the results of treatment with Agamree  after48 weeks in the VISION-DMD study, supporting the long-term efficacy and safety profile of the drug and concluding that it was generally well tolerated, consistent with the 24-week study findings.
Patients receiving Agamree at a dose of 6mg/kg per day showed maintenance of improvement for all motor outcomes to week 48, Santhera said, adding that those in the 2mg/kg per day cohort showed similar improvements as the higher dose for North Star Ambulatory Assessment after 48 weeks but less improvements for other motor outcomes.
Agamree also showed safety benefits for patients switching from standard-of-care corticosteroids in terms of bone health and growth
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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