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BioMarin's (BMRN) Dwarfism Drug Gets Approval in Europe

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BioMarin Pharmaceutical (BMRN - Free Report) announced that the European Commission (EC) has approved its once-daily injection, Voxzogo (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.

BioMarin’s shares were up 6% on Aug 27 in response to Voxzogo approval as it should boost BioMarin’s sales in future quarters. In the year so far, the stock price of BioMarin has declined 4.8% against the industry’s growth of 1%.

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Voxzogo, a C-type Natriuretic Peptide, is the first medicine in Europe to be approved 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 the base of the skull.

The approval in the EU was expected as in June, the European Medicines Agency's Committee for Medicinal Products for Human Use (“CHMP”) had given a positive opinion recommending the approval of Voxzogo. With the approval in the EU, Voxzogohas become eligible to treat more than 11,000 children affected by achondroplasia across Europe, Middle East and Africa.

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. The drug is expected to cost $300,000 per patient annually in France.

Meanwhile, in the United States, the new drug application (NDA) for the drug is under priority review 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 regulatory applications on vosoritideare based on final data from a phase III study, evaluating its efficacy and safety in children (aged 5-14), long-term safety and efficacy data from the ongoing phase II and phase III extension studies and extensive natural history data. 

Top-line data from the phase III study, announced previously, showed that cumulative height gain over the two-year treatment period was 3.52 cm more than the untreated children on treatment with vosoritide.

Additional studies are ongoing on vosoritide. A phase II study in achondroplasia patients aged 0 to 5 has completed enrollment.Also, 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.

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 from the biotech sector include Horizon Therapeutics (HZNP - Free Report) , Regeneron (REGN - Free Report) , and Repligen Corporation (RGEN - Free Report) . While Regeneron sports a Zacks Rank #1, Repligen and Horizon Therapeutics have a Zacks Rank of 2 (Buy).

Horizon Therapeutics’ earnings estimates have increased from $3.62 to $4.61 per share for 2021 and from $5.18 to $6.08 per share for 2022 in the past 30 days. The stock has risen 47.9% so far this year.

Regeneron’s earnings per share estimates have moved north from $49.96 to $54.15 for 2021 and from $40.91 to $44.11 for 2022 in the past 30 days. The stock has risen 37.3% so far this year.

Repligen’s earnings per share estimates have risen from $2.69 to $2.76 for 2021 and from $2.94 to $3.02 for 2022 in the past 30 days. The stock has risen 43.9% so far this year.