BioMarin's MAA for Dwarfism Candidate Gets EMA Validation
BioMarin Pharmaceutical Inc. BMRN announced that the European Medicines Agency (EMA) has accepted for review its marketing authorization application (MAA) for vosoritide. The company is seeking an approval for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for the treatment of children with achondroplasia, the most common form of dwarfism.
Last month, BioMarin filed the MAA for vosoritide to the EMA. The application was based on the final data from a phase III study evaluating the efficacy and safety of vosoritide, long-term safety and efficacy data from the ongoing phase II and phase III extension studies and an extensive natural history data.
BioMarin expects to file a regulatory application during the third quarter of 2020, seeking an approval for vosoritide in the United States.
Both the FDA and the EMA granted an Orphan Drug designation to vosoritide for the treatment of achondroplasia, for which no drug is approved yet.
Notably, an investigator-initiated study on vosoritide for a second indication — genetic short stature (GSS) — has begun, as part of a research collaboration with Children's National Hospital.
Shares of BioMarin have rallied 38.2% year to date compared with the industry’s growth of 3.9%.
Earlier this week, Ascendis Pharma ASND announced that the European Commission (EC) has granted an Orphan Designation to its candidate TransCon CNP for the treatment of achondroplasia. The company is currently evaluating TransCon CNP in the global phase II ACcomplisH study to address achondroplasia.
Meanwhile, BioMarin’s another key candidate, Roctavian/valrox, a gene therapy for severe hemophilia A, is also under review in the United States and Europe. A decision from the FDA is expected on Aug 21. If approved, Valrox is anticipated to be transformational as it has the potential to dramatically change the treatment paradigm. Four-year data from an ongoing phase I/II study demonstrated that all participants who received a single administration of Roctavian remained off exogenous factor VIII prophylactic therapy and demonstrated a greater than 90% reduction in bleeding episodes four years after treatment.
Zacks Rank & Stocks to Consider
BioMarin currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Emergent BioSolutions Inc. EBS and Horizon Therapeutics Public Limited Company HZNP, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Emergent’s earnings estimates have moved 38.9% and 78.8% north for 2020 and 2021, respectively, over the past 60 days. The stock has skyrocketed 150.1% year to date.
Horizon’s earnings estimates have been revised 74% and 60.1% upward for 2020 and 2021 each over the past 60 days. The stock has soared 100% year to date.
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