BioMarin Pharmaceutical Inc. ( BMRN Quick Quote BMRN - Free Report) announced that it has received positive recommendation from European Medicines Agency's Committee for Medicinal Products for Human Use (“CHMP”) for its investigational once-daily injection, vosoritide, to treat achondroplasia, a common form of dwarfism in children from the age of two until after puberty, when they attain final adult height.
Vosoritide is a once-daily injection analog of C-type Natriuretic Peptide to treat achondroplasia, the most common form of skeletal dysplasia that causes disproportionate short stature in humans and disordered architecture in the long bones, spine, face as well as base of the skull.
The CHMP’s recommendation is based on data from the phase III study evaluating the drug’s efficacy and safety. The primary endpoint of the two-year study was the change in growth velocity from baseline to treat achondroplasia in children with vosoritide compared to placebo. The company anticipates the approval from the European Commission (“EC”) in third-quarter 2021. Once approved by EC, the drug would be marketed under the trade name Voxzogo.
In the year so far, the stock price of BioMarin has declined 3.1% against the
industry’s growth rate of 0.5%. Image Source: Zacks Investment Research
Meanwhile, in the United States, the new drug application (NDA) for the drug is under priority review with the FDA with a PDUFA target date of November 2021. Once approved, the NDA may even qualify for a Priority Review Voucher. The FDA’s pre-approval inspection of BioMarin’s manufacturing facility of vosoritide drug substance was completed in first-quarter 2021.
The drug has also received a temporary authorization for use in France to begin immediate treatment under an authorized process, since there is no approved treatment for achondroplasia.
Per the company, once approved, vosoritide will become eligible to treat more than 11,000 children affected by achondroplasia across Europe, Middle East and Africa.
BioMarin has heavily invested in the R&D of vosoritide. This increase is on account of redirection of the amount allocated for sales and marketing of other programs to the R&D of vosoritide for supporting pre-commercialization activities related to the drug based on expected approval timelines. Pursuant to vosoritide’s potential approval, the company expects to generate blockbuster sales from the same.
BioMarin currently carries a Zacks Rank #3 (Hold). You can see
the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here .
Some better-ranked stocks in the medical sector include
BioNTech ( BNTX Quick Quote BNTX - Free Report) , Repligen ( RGEN Quick Quote RGEN - Free Report) and Bayer ( BAYRY Quick Quote BAYRY - Free Report) .
While BioNTech sports a Zacks Rank of #1, Repligen and Bayer both carry a Zacks Rank of #2 (Buy).
BioNTech’s earnings estimates for 2021 have increased from $18.39 to $30.85 per share in the past 60 days while that of 2022 has risen from $7.55 to $25.20 per share over the same period. The stock has surged 182.2% this year so far.
Repligen’s earnings estimates for 2021 have increased from $1.91 to $2.26 per share in the past 60 days while that of 2022 has risen from $2.23 to $2.56 per share over the same period.
Bayer’s earnings estimates for 2021 have increased from $1.74 to $1.81 per share in the past 60 days while that of 2022 has risen from $1.89 to $2.00 per share over the same period. The stock has risen 4.4% in the year so far.
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