FDA Grants Fast Track to Mavrix Bio's Gene Therapy for Angelman Syndrome
Mavrix Bio, a biotech company, has received fast track designation from the FDA for its gene therapy MVX-220, intended for treating Angelman syndrome. This decision reflects the urgent need for therapies for this condition and paves the way for accelerated clinical development and review.
MVX-220 is set to enter its first-in-human trial, ASCEND-AS, at Rush University Medical Center in Chicago. This Phase 1/2 study will enrol 12 participants, aged 4 to 50, with Angelman syndrome caused by different genetic mutations. The trial's primary focus is to assess the therapy's safety over the initial two years, with a follow-up period of five years.
The fast track designation allows for more frequent interactions with the FDA and potential eligibility for accelerated approval pathways. Mavrix's chief development officer, Allyson Berent, welcomed the decision, highlighting the unmet medical need for Angelman syndrome therapies. The company's gene therapy was developed with support from FAST and is now moving into clinical testing with Mavrix's launch earlier this year. Gemmabio, founded by gene therapy pioneer James M. Wilson, MD, PhD, supports Mavrix in its mission to bring gene-targeted therapies to patients sooner.
MVX-220, with its fast track designation, is poised to expedite the clinical development and review process for Angelman syndrome treatment. The upcoming ASCEND-AS trial marks a significant step towards evaluating the therapy's safety and efficacy, potentially offering hope to individuals living with this condition.
Read also:
- Americans Lose Insurance Under New Tax Legislation, Affecting 10 Million Citizens
- Pro-Life Group Condemns FDA's Approval of Generic Abortion Drug
- Trump Signs Law Defunding Planned Parenthood, Threatening Healthcare Access for Millions
- Historian Ute Frevert Explores Germans' Emotional Bond With Constitutions
