BioMarin Pharmaceutical Inc. Website

BioMarin Pharmaceutical Inc.

NasdaqGS-BMRN

Company Overview

BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops valoctocogene roxaparvovec, an adeno associated virus vector, which is in Phase III clinical trial for the treatment of patients with severe hemophilia A; BMN 307, an AAV5 mediated gene therapy, which is in Phase 1/2 clinical trial to normalize blood Phe concentration levels in patients with PKU; and BMN 255 that is in Phase 1/2 clinical trial for treating primary hyperoxaluria. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. The company was incorporated in 1996 and is headquartered in San Rafael, California.

  • Name

    BioMarin Pharmaceutical Inc.

  • CEO

    Alexander Hardy

  • Website

    www.biomarin.com

  • Sector

    Biotechnology

  • Year Founded

    1996

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Bulls Say

  • BioMarin's approved drugs have been granted orphan-drug status in the US and the EU, providing them with at least seven and 10 years of market exclusivity, respectively.

  • BioMarin's drugs target rare chronic conditions that often require treatment from a very young age, and while locating eligible patients on a global level is challenging, the firm has high patient retention rates.

  • With a growing portfolio in an attractive rare-disease niche and an acceleration of profit growth since 2022, BioMarin could be an acquisition target for pharmaceutical firms with pipelines to fill.

Bears Say

  • Diagnosis of rare genetic diseases can be difficult, and BioMarin could have a hard time locating enough patients to recoup development and manufacturing costs.

  • BioMarin's gene therapy pipeline has faced setbacks, including a three-year delay in the US approval of Roctavian and the stalled development of PKU gene therapy BMN 307, as the Food and Drug Administration halted a trial to investigate potential cancer risks.

  • Viral contamination forced Genzyme to halt manufacturing of its enzyme replacement therapies in 2009, highlighting the manufacturing risk in the industry.

Source: Morningstar Analysis - Oct 29, 2025

What's happening

Nov 12, 2025 - Dec 12, 2025

BioMarin Pharmaceutical Inc. Faces Challenges Amidst Strategic Shifts and Analyst Downgrades

  • BioMarin's stock declined by 2.3% over the past month, underperforming against the S&P 500's gain of 0.7%.
  • Leerink Partners downgraded BMRN from "Outperform" to "Market Perform," citing increased competition and concerns about product pipeline gaps.
  • Despite negative sentiment, analysts maintain a consensus rating of "Moderate Buy" on key therapies for rare diseases.

Over the last month, BioMarin Pharmaceutical Inc. (BMRN) experienced a decline of 2.3%, which is notably lower than the S&P 500’s increase of 0.7%. This downturn was significantly influenced by bearish developments that affected investor sentiment and stock valuation. A critical factor in this performance was Leerink Partners' downgrade of BMRN from "Outperform" to "Market Perform," with a revised price target set at $60 due to increasing competition in the achondroplasia market and concerns regarding gaps in BioMarin's product pipeline.

The company's recent strategic decisions also contributed to investor unease. Workforce reductions alongside the discontinuation of the Roctavian program were perceived as efforts to strengthen long-term positioning; however, these moves raised immediate concerns about operational impacts and competitive standing within its sector. Nevertheless, analysts continue to express cautious optimism with a consensus rating of "Moderate Buy," particularly for BMRN’s therapies targeting rare diseases such as Vimizim and Naglazyme.

In early December, mixed earnings expectations led analysts to adjust their target prices for BMRN shares amid projections estimating earnings at approximately $3.15 per share with an anticipated revenue growth rate of only 4.1% year-over-year—reflecting tempered optimism influenced by broader market dynamics affecting biotech stocks.

On a more positive note during this period, BioMarin announced participation in significant industry events like the Jefferies Global Healthcare Conference scheduled for November 18th; this announcement initially bolstered investor confidence as it underscored their commitment to innovation through ongoing clinical developments related to rare genetic conditions.

Overall, while some bullish indicators emerged from conference participation and analyst ratings suggesting potential upside opportunities exist within specific therapies, they were overshadowed by critical downgrades and strategic shifts leading to overall underperformance against both sector benchmarks and broader market indices—specifically underperforming relative to Health Care (XLV) sector by -4.2%.

NasdaqGS:BMRN