RARE Daily

FDA Awards Grant to ReveraGen and Santhera to Fund Trial

September 27, 2021

The U.S. Food and Drug Administration has made a $1.2 million grant to ReveraGen Biopharma and Santhera Pharmaceuticals to support the clinical study of the experimental therapy vamorolone in adults and children with the progressive muscle wasting disease Becker muscular dystrophy.

Photo: Paula Clemens, professor of Neurology at the University of Pittsburgh School of Medicine

The funding, made under the FDA’s Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases grants program, adds to existing grants from the National Institutes of Health, NIAMS, and the Foundation to Eradicate Duchenne for the study.

Becker muscular dystrophy (BMD) is a serious, progressively debilitating, and potentially fatal inherited disorder. BMD results from truncation or mutation of the X-linked dystrophin gene yielding unstable and/or dysfunctional dystrophin expression in muscles. Individuals with BMD, typically males, have ongoing muscle fiber degeneration that eventually leads to fibrosis, progressive loss of skeletal muscle function, and that can lead to severe disability and early death.

“There are currently no approved drugs for BMD in any country, and there is a high unmet need,” said Paula Clemens, professor of Neurology at the University of Pittsburgh School of Medicine, and co-principal investigator on the FDA, NIH, and Foundation awards.

Vamorolone is a first-in-class drug candidate that binds to the same receptor as corticosteroids but modifies its downstream activity and as such is a dissociative partial agonist. This mechanism has the potential to dissociate efficacy from typical steroid safety concerns and therefore vamorolone could emerge as a promising alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is substantial unmet medical need in many patient groups where corticosteroids are standard of care or hold therapeutic promise, but side effects limit prescription and patient adherence.

Vamorolone has been granted Orphan Drug status in the United States and in Europe for DMD and has received Fast Track and Rare Pediatric Disease designations by the FDA and Promising Innovative Medicine status from the European Medicines Agency and United Kingdom’s Healthcare Products Regulatory Agency for Duchenne muscular dystrophy.

Vamorolone has shown retention of efficacy and reduction of safety concerns typically associated with corticosteroids in Duchenne muscular dystrophy. It will now be tested in a 24-week clinical exploratory trial in Becker muscular dystrophy. The double-blind trial will test efficacy and safety of daily vamorolone on motor outcomes and established biomarker outcomes, with participants randomized 2:1 vamorolone or placebo. The clinical trial plans to enroll at sites in Padova, Italy and Pittsburgh.

“While the drug development pipeline has greatly expanded for DMD in recent years, there are very few clinical investigational efforts underway for BMD,” said Elena Pegoraro, professor of Neurology at the University of Padova in Italy. “Corticosteroids are often not tolerated by patients with BMD due to their side effects. Therefore, the lessened side effect burden of vamorolone seen in DMD trials may prove important to the underserved BMD patient community.”

Author: Rare Daily Staff

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