Skip to content

FDA Grants Orphan Drug Designation to Sarconeos for Duchenne Muscular Dystrophy

May 16, 2018

May 15, 2018

Today, BIOPHYTIS announced that the US Food and Drug administration (FDA) has granted orphan drug designation to its drug candidate, Sarconeos, for Duchenne Muscular Dystrophy (DMD).

In a recent comment, Stanislas Veillet, CEO of BIOPHYTIS, stated: “The orphan drug designation that has just been granted by the FDA is a significant milestone for our team and the development of Sarconeos in Duchenne Myopathy, a particularly severe pediatric indication. We hope to obtain this designation in Europe as well in the coming weeks. We will then be able to conduct the MYODA clinical program. This program will consist of two studies: a pharmacokinetic phase 1/2a MYODA-PK study, which could begin in 2018 and an efficacy phase 2/3 MYODA-INT study, which could start in 2019.”

Sarconeos activates the MAS receptor (major player of the renin-angiotensin system), restoring muscular anabolism and inhibiting myostatin as a result. This action has demonstrated meaningful activity in animal models of muscular dystrophies.

In 2017, Sarconeos’ proof-of-concept in the treatment of DMD was presented at the World Muscle Society. In the reference animal model of DMD, it was exhibited that Sarconeos significantly improved exercise tolerance and muscle strength and reduced muscle fibrosis.

In comparison with the development of Sarcopenia, orphan drug designation is BIOPHYTIS’ first regulatory step in obtaining authorizations to develop Sarconeos in DMD. BIOPHYTIS’ drug-paring candidates designate the implementation of a dual development strategy for genetic degenerative diseases in addition to chronic geriatric diseases.

Additionally, 2 main clinical studies compose Sarconeos’ clinical development plan in DMD myopathy: a pharmacokinetic phase I/II MYODA-PK study to be initiated in 2018 and a phase II/III efficacy study, MYODA-INT, that could commence in 2019. Since very few and effective treatment options currently exist for DMD, Sarconeos has the potential to significantly stall the progression of the disease. It also has the potential to be used as a standalone treatment, or it could be used in combination with gene therapy when it is available for children with DMD myopathy.

In Europe, BIOPHYTIS recently filed an application for an orphan drug designation for Sarconeos in DMD myopathy with the EMA (European Medicines Agency). A response is anticipated in the coming weeks.