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Here's Why You Should Invest in BioMarin (BMRN) Stock Now

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BioMarin Pharmaceutical’s (BMRN - Free Report) dwarfism drug Voxzogo is driving the company’s top line on the back of strong demand. The recent label expansion to Voxzogo for use in infants with achondroplasia is likely to boost sales further in the days ahead.

Meanwhile, after several delays, the FDA approved BMRN’s Roctavian, a gene therapy for treating hemophilia A last June. The company also has an attractive early-stage pipeline with a focus on gene therapies.

Here are some reasons why investors should consider betting on BioMarin stock.

Good Rank and Rising Estimates: BioMarin currently has a Zacks Rank #2 (Buy).

In the past 60 days, the Zacks Consensus Estimate for BioMarin’s 2024 earnings per share has improved from $2.78 to $3.16. The same for 2025 has moved up from $3.66 to $3.94.

Shares of BioMarin have gained 3.5% in the past six months against the industry’s decline of 1.1%.


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Voxzogo - A Key Sales Driver: Voxzogo is the first medicine to be approved in the United States and Europe for treating achondroplasia, the most common form of dwarfism. A progressive condition, achondroplasia occurs in one in every 25,000 newborns worldwide. Voxzogo can fulfill the need by targeting the root cause of the disease.

Voxzogo has been witnessing rapid uptake driven by strong prescription demand. BioMarin is seeking approvals for the drug across multiple new markets, which can boost sales. Voxzogo generated sales worth $336.7 million in the first half of 2024, up 67% year over year, mainly driven by new patient initiations.

BMRN started a pivotal registrational program in 2023 on Voxzogo for a new potential second indication — hypochondroplasia — a condition characterized by impaired bone growth. Management is also preparing to initiate additional clinical programs in 2024 evaluating Voxzogo in idiopathic short stature, Noonan syndrome, Turner syndrome and SHOX deficiency.

Focus on Gene Therapy Space Encouraging: BioMarin is one of the few companies that has shown encouraging progress in the gene therapy space and commercially markets a gene therapy treatment. Roctavian is the first and only one-shot gene therapy approved for treating adults with severe hemophilia A in the United States and Europe. A rare genetic blood disorder, hemophilia A, is caused by a missing or defective clotting protein, FVIII, which prevents normal blood clotting. Roctavian has been designed to deliver a functional gene that enables the body to produce the FVIII protein on its own, reducing the need for ongoing prophylaxis.

Roctavian generated $7.4 million in sales in the second quarter of 2024 compared with $0.8 million in the previous quarter. BioMarin is actively working to introduce new gene therapies in its portfolio.

Other Pipeline Candidates Show Promise: After completing a strategic review of its pipeline, BioMarin has decided to advance the development of three early-stage pipeline candidates. These include BMN 351, a next-generation oligonucleotide, for treating Duchenne Muscular Dystrophy. Per management, BMN 351 can be administered chronically and produces near full-length dystrophin, giving it an edge even over currently approved gene therapies. An early-stage study is ongoing with data from the same expected by 2024-end.

The company is also planning to conduct a first-in-human study on BMN 349, an oral therapeutic for treating alpha-1 antitrypsin deficiency-associated liver disease, later in 2024. BMRN is planning to advance BMN 333, a long-acting C-type natriuretic peptide as a potential treatment for multiple growth-related conditions. Clinical studies are expected to start in early 2025.

Other Stocks to Consider

Some other top-ranked stocks in the biotech sector are Immatics N.V. (IMTX - Free Report) , Fulcrum Therapeutics, Inc. (FULC - Free Report) and Entrada Therapeutics, Inc. (TRDA - Free Report) , each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Immatics’ 2024 loss per share have narrowed from $1.26 to $1.25. Loss per share estimates for 2025 have narrowed from $1.49 to $1.41. Year to date, shares of IMTX have risen 8%.

IMTX’s earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 32.57%.

In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from $1.24 to 48 cents. Loss per share estimates for 2025 have narrowed from $1.71 to $1.51. Year to date, shares of FULC have increased 33.5%.

FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.

In the past 60 days, estimates for Entrada Therapeutics’ 2024 loss per share have narrowed from 14 cents to 13 cents. Loss per share estimates for 2025 have narrowed from $3.44 to $3.21. Year to date, shares of TRDA have lost 5.9%.

TRDA’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 42.18%.

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