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CHMP Endorses BioMarin's (BMRN) Voxzogo for Use in Infants

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BioMarin (BMRN - Free Report) announced that the European Medicines Agency's (“EMA”) Committee for Medicinal Products for Human Use (“CHMP”) has given a positive opinion recommending label expansion for Voxzogo (vosoritide) to treat achondroplasia, the most common form of dwarfism, in children aged four months and above.

This recommendation is based on data from a phase II study on Voxzogo in children aged between four months and five years with achondroplasia. Data from the study showed that treatment with the drug showed an improvement in height Z-score of approximately 0.3 standard deviations across all age groups after one year of treatment.

A final decision from the European Commission is expected before the end of this year.

Voxzogo is currently approved in the European Union for children with achondroplasia aged two years and above, with open growth plates.

The drug is also approved for a similar indication in the United States but for use in children five years and older. The company’s supplemental new drug application (sNDA) is under the FDA’s review, seeking approval to treat the disease in children under five years old. A final decision is expected next month.

In the year so far, BioMarin’s shares have lost 14.8% compared with the industry’s 14.1% decline.

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Since its launch, Voxzogo has seen rapid uptake driven by solid prescription demand. BioMarin is seeking approvals for the drug across multiple new markets, which can boost drug sales.

During first-half 2023, Voxzogo generated sales worth $201 million, mainly driven by new patient initiations, which is encouraging for a newly launched drug. For 2023, management expects to record Voxzogo sales of $400 million to $440 million, compared to $169 million recorded as revenues in 2022.

Currently, Voxzogo is the first and only medicine for achondroplasia. If the therapy were to be approved for these two age groups in the United States and Europe, more than a thousand children suffering from achondroplasia would be eligible for treatment. This will likely boost BioMarin’s topline.

Voxzogo will likely face stiff competition from BridgeBio Pharma (BBIO - Free Report) , which is also planning to advance its pipeline drug for treating achondroplasia. In March, BridgeBio reported encouraging data from a cohort of the phase II PROPEL2 study wherein patients with achondroplasia treated with BridgeBio’s infigratinib achieved a significant and robust increase in annualized height velocity (“AHV”) at six months. The reported results were noted to have been a substantial improvement over treatment with Voxzogo.

Earlier this month, BridgeBio announced that it has received positive feedbacks from both the EMA and FDA on the design for a pivotal phase III study (PROPEL3) on infigratinib in children (aged 3 to 18 years) with achondroplasia. This study is expected to start before 2023-end.


Zacks Rank & Stocks to Consider

BioMarin currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include Precision BioSciences (DTIL - Free Report) and Selecta Biosciences , each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 30 days, estimates for Precision BioSciences’ 2023 loss per share have improved from 82 cents to 77 cents. During the same period, the loss estimates per share for 2024 have narrowed down from 48 cents to 31 cents. Year to date, shares of DTIL have lost 65.6%.

Earnings of Precision BioSciences beat estimates in two of the trailing four quarters while beating the mark on other two occasions, delivering an average earnings surprise of 20.20%. In the last reported quarter, Precision BioSciences’ earnings beat estimates by 56.52%.

In the past 30 days, estimates for Selecta Biosciences’ 2023 loss per share have improved from 39 cents to 33 cents. During the same period, the estimates per share for 2024 narrowed down from 35 cents to 7 cents. Year to date, shares of Selecta have risen 0.9%.

Earnings of Selecta Biosciences beat estimates in two of the trailing four quarters and missed in the remaining two, the average negative surprise being 47.57%. In the last reported quarter, Selecta Biosciences’ earnings beat estimates by 22.22%.

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