The U.S. Food and Drug Administration (FDA) has agreed to review a Biologics License Application (BLA) submitted by Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) for its recombinant human beta-glucuronidase (rhGUS). The submission of the BLA was alongside that of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), which was also accepted for review.
The FDA granted Priority Review status to RaRE’s BLA for rhGUS, an investigational candidate under development for the treatment of mucopolysaccharidosis VII (MPS VII) otherwise known as Sly syndrome. The company’s BLA A PDUFA action date has now been scheduled for Nov 16, 2017. On the other hand, the acceptance of MAA by the EU awaits a judgment from the Committee for Medicinal Products for Human Use (CHMP), which should not be later than the first half of 2018.
The regulatory filings were supported by data from a phase III study
The acceptance and review of BLA and MAA did not just happen. It involved a phase III study, conducted at four sites in the U.S. and was primarily evaluating the safety and efficacy of rhGUS. The investigation subjects were 12 patients within 5–35 years age bracket and those who suffered from MPS 7 – a rare lysosomal storage disease. A 4 mg/kg of rhGUS was administered to them every other week for about 48weeks.
The results were compelling because they demonstrated a 64.8% of rapid and sustained reduction baseline in urinary GAG excretion. Surprisingly this was after 24 weeks of treatment. Apart from proving to have met the endpoint, the results also showed evidence of clinical improvement with mild to moderate adverse events.
Let’s explore Ultragenyx
Since its inception in 2010, the clinical-stage biopharmaceutical company has been building a varied portfolio of product candidates. With a lot of commitment, it has been bringing the market novel products for the treatment of rare and ultra-rare diseases. Its main goal is in the delivery of safe and effective therapies to patients.
The company’s Chief Executive Officer and President Emil D. Kakkis confirms, “We are pleased that the FDA has granted priority review to the rhGUS BLA and are looking forward to working with both the FDA and EMA in the coming months”
Meanwhile, Ultragenyx’s stock was trading at $53.01 a fall of $2.90 or -5.19%