UK MHRA assigns PIM status to ReveraGen’s Vamorolone as a DMD therapy
Category: #health  By Mateen Dalal  Date: 2019-10-22
  • share
  • Twitter
  • Facebook
  • LinkedIn

UK MHRA assigns PIM status to ReveraGen’s Vamorolone as a DMD therapy

Duchenne muscular dystrophy (DMD) approximately affects 1 in every 3,500 male births every year in the world. several biopharmaceutical and biotechnology firms across the globe are increasingly working towards the discovery and development of novel therapies for this disease.

One of the contributors to this effort is Santhera Pharmaceuticals, a renowned Swiss pharmaceutical firm focused at the commercialization and development of medicines for DMD (Duchenne muscular dystrophy), reportedly announced that ReveraGen Biopharma, a US based pharmaceutical firm, has been awarded the PIM (Promising Innovative Medicine) designation for their vamorolone drug by the UK MHRA (Medicines and Healthcare products Regulatory Agency) to treat DMD (Duchenne muscular dystrophy).

CEO of Santhera, Thomas Meier, PhD stated that Santhera congratulates ReveraGen on its success in finding a possible treatment for DMD and the company is thrilled about the PIM designation grant as it validates the capabilities of vamorolone as an advanced treatment approach which would address the high unmet medical requirement in young patients suffering from DMD.

The PIM designation granted by MHRA signifies that the agency considers vamorolone as a hopeful candidate for EAMS (Early Access to Medicines Scheme). In the United Kingdom, PIM is the primary step of EAMS, which aims to provide patients with seriously debilitating or life-threatening conditions access to those medicines which don’t have a marketing authorization yet if there is a strong unmet medical need.     

ReveraGen’s Vamorolone is a steroidal, anti-inflammatory first-in-class investigational drug that is under development as a DMD treatment. Data from clinical and non-clinical studies specified that treatment with vamorolone showed a persistent improvement of muscle function along with lesser adverse effects, generally reported for traditional corticosteroids.

Vamorolone drug has received Orphan Drug status in Europe and the U.S. Furthermore, the drug has also received designations of Fast Track and Rare Pediatric Disease by the U.S. FDA.     

The currently under progress Phase IIb VISION-DMD 48 week trial (VBP15-004; NCT03439670) is specifically designed like a pivotal study to showcase safety and efficacy of vamorolone in comparison to placebo and prednisone in 120 boys aged 4 to below 7 suffering from DMD who have not been treated with corticosteroids yet.

Source credit: http://www.santhera.com/assets/files/press-releases/2019-10-21_PIM_Vamorolone_e_final.pdf

 

  • share
  • Twitter
  • Facebook
  • LinkedIn


About Author

Mateen Dalal    

Mateen Dalal

Mateen has completed his Bachelor’s degree in electronics and telecommunication engineering, post which he lent his proficiency to the industry, working as a quality and test engineer. Drawn intricately toward the field of content creation however, Mateen soon switc...

Read More

More News By Mateen Dalal

Gennova begins enrollment for Phase 1/2 trials of mRNA COVID vaccine
Gennova begins enrollment for Phase 1/2 trials of mRNA COVID vaccine
By Mateen Dalal

India's first mRNA-based COVID-19 vaccine, HGCO19, formulated by Pune-based biotech firm, Gennova Biopharmaceuticals is about to enter its Phase 1/2 clinical trials with the enrollment of candidates, said the Department of Biotechnology (DBT). R...

Mashcor launches new independent search engine for the Afrotech sector
Mashcor launches new independent search engine for the Afrotech sector
By Mateen Dalal

Mashcor, a leading South African digital agency, has reportedly announced the launch of Africa’s first indigenous search engine. The new search engine has been specifically designed to connect the region’s rapidly expanding tech sector to...

Canada and Germany ink new energy partnership with emphasis on hydrogen
Canada and Germany ink new energy partnership with emphasis on hydrogen
By Mateen Dalal

The government of Canada has inked a new, comprehensive energy partnership deal with the government of Germany to set up a formal collaboration across a wide array of shared energy priorities, including hydrogen and LNG. According to reports, this c...