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BioMarin's (BMRN) Dwarfism Drug Gets FDA's Nod, Stock Up
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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the FDA has granted approval to Voxzogo (vosoritide) injection to treat achondroplasia, a common form of dwarfism in children.
Voxzogo has been approved to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates). The accelerated approval by the FDA is based on an improvement in annualized growth velocity. BioMarin needs to conduct a post-marketing study to get final approval for the drug.
BioMarin’s shares were up 10.3% on Nov 19 in response to Voxzogo approval in the United States. Voxzogo’s FDA approval opens up a key sales opportunity for BioMarin and restores investors’ confidence in the stock. In the year so far, the stock price of BioMarin has risen 4.3% against the industry’s decline of 14.2%.
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Voxzogo, a C-type natriuretic peptide, is the first medicine in the United States 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. There is an urgent unmet medical need for this progressive condition, which occurs in one in every 25,000 newborns worldwide. Voxzogo can potentially fulfill this need by targeting the root cause of the disease.
Voxzogo is expected to be available in the United States by mid-to-late December. The FDA issued a Rare Pediatric Disease Priority Review Voucher (PRV) along with the approval. BioMarin can use the PRV to get priority review to a subsequent drug application that would not otherwise qualify for priority review.
Voxzogo was approved in Europe in August. In the brief period since its launch, the drug generated sales worth $0.1 million. On the third-quarter conference call held last month, BioMarin said that the level of initial prescription demand for patients seeking Voxzogo treatment in Europe exceeded its expectations.
The regulatory applications were based on final data from a phase III study, evaluating the efficacy and safety of vosoritide in children (aged 5-14). 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.
Some better-ranked stocks from the biotech sector include Sarepta Therapeutics (SRPT - Free Report) , Editas Medicine (EDIT - Free Report) and IVERIC bio , all having a Zacks Rank of 2 (Buy).
Loss per share estimates for Sarepta have narrowed from $6.95 to $4.99 for 2021 and from $4.83 to $3.61 for 2022 in the past 30 days. Sarepta delivered an earnings surprise of 11.06%, on average, in the last four quarters.
Editas Medicine’s loss estimates have narrowed from $3.55 to $3.16 per share for 2021 and from $3.63 to $3.47 per share for 2022 in the past 30 days. Editas Medicine delivered an earnings surprise of 0.83%, on average, in the last four quarters.
IVERIC bio’s stock has risen 129.6% this year so far. Loss per share estimates for IVERIC bio have narrowed from $1.18 to $1.12 per share for 2021 and from $1.17 to $1.10 per share for 2022 in the past 30 days.
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BioMarin's (BMRN) Dwarfism Drug Gets FDA's Nod, Stock Up
BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the FDA has granted approval to Voxzogo (vosoritide) injection to treat achondroplasia, a common form of dwarfism in children.
Voxzogo has been approved to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates). The accelerated approval by the FDA is based on an improvement in annualized growth velocity. BioMarin needs to conduct a post-marketing study to get final approval for the drug.
BioMarin’s shares were up 10.3% on Nov 19 in response to Voxzogo approval in the United States. Voxzogo’s FDA approval opens up a key sales opportunity for BioMarin and restores investors’ confidence in the stock. In the year so far, the stock price of BioMarin has risen 4.3% against the industry’s decline of 14.2%.
Voxzogo, a C-type natriuretic peptide, is the first medicine in the United States 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. There is an urgent unmet medical need for this progressive condition, which occurs in one in every 25,000 newborns worldwide. Voxzogo can potentially fulfill this need by targeting the root cause of the disease.
Voxzogo is expected to be available in the United States by mid-to-late December. The FDA issued a Rare Pediatric Disease Priority Review Voucher (PRV) along with the approval. BioMarin can use the PRV to get priority review to a subsequent drug application that would not otherwise qualify for priority review.
Voxzogo was approved in Europe in August. In the brief period since its launch, the drug generated sales worth $0.1 million. On the third-quarter conference call held last month, BioMarin said that the level of initial prescription demand for patients seeking Voxzogo treatment in Europe exceeded its expectations.
The regulatory applications were based on final data from a phase III study, evaluating the efficacy and safety of vosoritide in children (aged 5-14). 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.
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 Sarepta Therapeutics (SRPT - Free Report) , Editas Medicine (EDIT - Free Report) and IVERIC bio , all having a Zacks Rank of 2 (Buy).
Loss per share estimates for Sarepta have narrowed from $6.95 to $4.99 for 2021 and from $4.83 to $3.61 for 2022 in the past 30 days. Sarepta delivered an earnings surprise of 11.06%, on average, in the last four quarters.
Editas Medicine’s loss estimates have narrowed from $3.55 to $3.16 per share for 2021 and from $3.63 to $3.47 per share for 2022 in the past 30 days. Editas Medicine delivered an earnings surprise of 0.83%, on average, in the last four quarters.
IVERIC bio’s stock has risen 129.6% this year so far. Loss per share estimates for IVERIC bio have narrowed from $1.18 to $1.12 per share for 2021 and from $1.17 to $1.10 per share for 2022 in the past 30 days.