BPG BLOG (58)

FDA Grants Sanofi and Sobi Factor VIII Therapy Breakthrough Designation
by BPG Blog

The FDA granted Sanofi and Sobi’s investigational blood-clotting drug efanesoctocog alfa a Breakthrough Therapy designation for treatment of patients with hemophilia A.

Efanesoctocog alfa is a novel recombinant factor VIII therapy designed to extend protection from bleeds with a once-weekly prophylactic dose.

The breakthrough designation was supported by phase 3 data showing that the treatment helped prevent bleeding in individuals with the rare, life-threatening genetic bleeding disorder over a 52-week period.

The FDA granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021.

Click here to read the full article at FDANews

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (50)

PTC’s first gene therapy gains European regulator’s backing
by BPG Blog

The recommendation from the EMA’s human drugs committee is good news for the gene therapy field after a string of negative developments. Clinical and regulatory setbacks have weighed on company valuations, limiting their fundraising options and leading to restructuring and layoffs for a number of gene therapy developers.

PTC’s regulatory decision is the first of several expected in the coming months that, if positive, could help turn investors’ views around. After its European disappointment, Bluebird is expecting Food and Drug Administration decisions on its two gene therapies this year, while closely watched projects from BioMarin Pharmaceutical and UniQure could also be under FDA review soon. Likewise, PTC expects by the end of September to ask the FDA for approval of Upstaza.

PTC’s therapy corrects a mutation that disrupts production of an enzyme called aromatic L-amino acid decarboxylase, which is in turn vital to production of other chemicals needed for nervous system signaling. As with Luxturna and Zolgensma, Upstaza uses an adeno-associated virus to deliver the corrected gene into cells.

Click here to read the full article at Biopharma Dive

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (27)

Ocugen Sees Ray of Light with Gene Therapy for Retinitis Pigmentosa
by BPG Blog

Ocugen, which has faced a string of delays with its Bharat Biotech-partnered vaccine, reported some good news today.

The company, based in Malvern, Pennsylvania, reported that the independent Data and Safety Monitoring Board (DSMB) involved in the Phase I/II trial of OCU400, a gene therapy in development for Retinitis Pigmentosa (RP), recommended the study proceed with enrolling more patients. The trial is evaluating the modifier gene therapy OCU400 for RP caused by mutations in the nuclear receptor subfamily 2 group E member 3 (NR2E3) and Rhodopsin (RHO) genes. Ocugen recently dosed the first patient in the study. The DSMB recommended the company continue enrolling the remaining subjects in the current cohort at the target dose level.

RP is a family of rare eye diseases affecting the retina. RP results in the retina breaking down over time, causing vision loss. Symptoms typically begin in childhood, but the majority of patients eventually lose most of their vision. The most common early symptom is loss of night vision.

Click here to read the full article at BioSpace

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.