Vamorolone Designated Promising Innovative Medicine in UK for DMD Treatment

Marta Figueiredo, PhD avatar

by Marta Figueiredo, PhD |

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The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to ReveraGen’s vamorolone for the treatment of Duchenne muscular dystrophy (DMD).

PIM designation indicates that the MHRA considers vamorolone likely to offer major therapeutic advantages for DMD patients. The MHRA will next assess the potential benefits and risks of vamorolone, based on its clinical studies.

This decision means that vamorolone, a potential oral therapy, is a promising candidate for the U.K.’s early access to medicines scheme (EAMS). EAMS seeks to help people with serious or life-threatening conditions gain access to promising therapy candidates for disorders with few available medical treatments.

“We congratulate ReveraGen on this success and are excited about the PIM designation as it further validates the potential of vamorolone as an innovative treatment approach addressing the high unmet medical need in young patients with DMD,” Thomas Meier, PhD, CEO of Santhera Pharmaceuticals, which owns sub-license rights to vamorolone, said in a press release.

Vamorolone (or VBP15) is an anti-inflammatory steroid treatment designed to slow DMD progression without the severe systemic side effects associated with corticosteroids, such as prednisone and Emflaza (deflazacort, by PTC Therapeutics). Although it binds to the same receptors as corticosteroids, vamorolone leads to different cellular effects, thereby representing a way to avoid the safety concerns of these standard treatments.

ReveraGen has opened a randomized, double-blind Phase 2b trial (NCT03439670) called VISION-DMD to compare its lead compound with prednisone or a placebo in boys with DMD.

The company expects to enroll 120 boys with DMD, 4 to 6 years old, who had never been treated with standard corticosteroids. Boys are being recruited at approximately 30 sites across the U.S., Canada, Europe, Australia, and Israel. More information on locations and contacts here.

Participants’ motor function will be assessed through several efficacy measures after 24 weeks of treatment. Boys on prednisone or placebo will then switch to vamorolone, with treatment ongoing for 20 more weeks. VISION-DMD is expected to be completed in May 2020.

Studies in mouse models of DMD showed that vamorolone improves skeletal and heart muscle health without the side effects of current corticosteroids.

Then, Phase 1 (NCT02415439) and Phase 2 clinical trials (NCT02760264; NCT02760277) showed that vamorolone leads to significant motor improvements in boys with DMD, while also supporting its better safety profile compared with cortiscosteroids.

Topline data from a two-year extension Phase 2 trial (NCT03038399) recently confirmed these efficacy and safety advantages in 23 boys with DMD after at least 18 months of treatment with vamorolone (2.0 or 6.0 mg/kg).

Vamolorone also has received orphan drug status in the U.S. and in Europe, as well as fast track and rare pediatric disease designations by the U.S. Food and Drug Administration.

These designations are expected to provide regulatory support and financial benefits to accelerate the clinical development and review of vamolorone, and to ensure marketing exclusivity for a period of time upon regulatory approval (seven years in the U.S. and 10 years in Europe).