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BioMarin shares five-year Pompe disease drug data at muscle congress

Fri, 02 Oct 2026 11:00:20 -0400

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) presented five-year Phase 3 open-label extension data for its two-drug combination Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) in late-onset Pompe disease at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan.

The analysis drew from 82 individuals who received Pombiliti and Opfolda continuously from the baseline of the initial PROPEL study. The group included 62 enzyme replacement therapy (ERT)-experienced patients, whose mean prior ERT duration at baseline was 7.6 years, and 20 ERT-naïve patients.

In the ERT-experienced cohort at year five, the mean change from baseline in percent predicted six-minute walk distance (pp6MWD) was

BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress

Fri, 02 Oct 2026 11:00:00 -0400

Long-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease

SAN RAFAEL, Calif., Oct. 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of POMBILITI^® (cipaglucosidase alfa-atga) + OPFOLDA^® (miglustat) in late-onset Pompe disease (LOPD) were presented at the 31st Annual Congress of the World Muscle Society

BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress

Fri, 02 Oct 2026 11:00:00 -0400

Long-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease

Form 4 BIOMARIN PHARMACEUTICAL For: Sep 30 Filed by: Friberg Gregory R

Thu, 01 Oct 2026 16:23:00 -0400

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Form 8-K BIOMARIN PHARMACEUTICAL For: Sep 24

Wed, 30 Sep 2026 16:16:00 -0400

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BioMarin Pharmaceutical Inc. (BMRN) PT Lowered to $97 at BMO Capital

Wed, 16 Sep 2026 08:41:40 -0400

(Updated - September 16, 2026 8:41 AM EDT)

BMO Capital analyst David Lebowitz lowered the price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to $97.00 (from $98.00) while maintaining a Outperform rating.

The analyst commented: "Noonan Exit Sharpens Voxzogo Focus on HCH/ISS With Limited Impact; BioMarin discontinued development of Voxzogo in Noonan syndrome, sharpening its focus on larger expansion opportunities in HCH and ISS. The decision to discontinue the Phase 2 Noonan study was unrelated to the safety or efficacy profile of Voxzogo. Relatively small addressable population of NS provides modest peak-sales opportunity, and impact from the discontinuation is therefore limited. We

BioMarin Pharmaceutical Inc. (BMRN) PT Lowered to $97 at BMO Capital

Wed, 16 Sep 2026 08:41:40 -0400

(Updated - September 16, 2026 8:41 AM EDT)

BMO Capital analyst David Lebowitz lowered the price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to $97.00 (from $98.00) while maintaining a Outperform rating.

BioMarin drops VOXZOGO development for Noonan syndrome children

Tue, 15 Sep 2026 08:34:15 -0400

BioMarin Pharmaceutical Inc. announced on September 15, 2026, that it will discontinue development of VOXZOGO for children with Noonan syndrome.

According to a company statement, the decision was based on an assessment of factors including study feasibility and the current treatment landscape for children with Noonan syndrome. BioMarin stated the decision was not related to the safety or efficacy of VOXZOGO.

BioMarin drops VOXZOGO development for Noonan syndrome children

Tue, 15 Sep 2026 08:34:15 -0400

BioMarin Pharmaceutical Inc. announced on September 15, 2026, that it will discontinue development of VOXZOGO for children with Noonan syndrome.

BioMarin drops VOXZOGO development for Noonan syndrome children

Tue, 15 Sep 2026 08:34:15 -0400

BioMarin Pharmaceutical Inc. announced on September 15, 2026, that it will discontinue development of VOXZOGO for children with Noonan syndrome.

BioMarin drops VOXZOGO development for Noonan syndrome children

Tue, 15 Sep 2026 08:34:15 -0400

BioMarin Pharmaceutical Inc. announced on September 15, 2026, that it will discontinue development of VOXZOGO for children with Noonan syndrome.

Form 8-K BIOMARIN PHARMACEUTICAL For: Sep 15

Tue, 15 Sep 2026 08:32:00 -0400

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Form 4 BIOMARIN PHARMACEUTICAL For: Sep 10 Filed by: Plenge Robert M

Mon, 14 Sep 2026 18:55:00 -0400

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Form 3 BIOMARIN PHARMACEUTICAL For: Sep 10 Filed by: Plenge Robert M

Mon, 14 Sep 2026 18:48:00 -0400

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Form 8-K BIOMARIN PHARMACEUTICAL For: Sep 10

Mon, 14 Sep 2026 09:26:00 -0400

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BioMarin adds Bristol Myers Squibb research chief to its board

Mon, 14 Sep 2026 09:05:20 -0400

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) has appointed Robert Plenge, M.D., Ph.D., to its board of directors, effective September 10, 2026, according to a company statement.

Dr. Plenge currently serves as Executive Vice President, Chief Research Officer and Head of Research at Bristol Myers Squibb, where he oversees scientific operations across nine research sites. He joined Bristol Myers Squibb in November 2019 as part of its acquisition of Celgene Corp., where he had led the immunology and inflammation portfolio in research and early development.

Prior to Celgene, Dr. Plenge was vice president and head of translational medicine at Merck & Co. He also

BioMarin Announces Appointment of Robert Plenge, M.D., Ph.D., Chief Research Officer of Bristol Myers Squibb, to Board of Directors

Mon, 14 Sep 2026 09:05:00 -0400

SAN RAFAEL, Calif., Sept. 14, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that the company has appointed Robert Plenge, M.D., Ph.D., to the company's Board of Directors, effective September 10, 2026.

BioMarin Announces Appointment of Robert Plenge, M.D., Ph.D., Chief Research Officer of Bristol Myers Squibb, to Board of Directors

Mon, 14 Sep 2026 09:05:00 -0400

H.C. Wainwright Reiterates Neutral Rating on BioMarin Pharmaceutical Inc. (BMRN)

Thu, 10 Sep 2026 06:50:17 -0400

H.C. Wainwright analyst Mitchell Kapoor reiterated a Neutral rating and $60.00 price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN).

The analyst commented: "YUVIWEL's 52-dose infant schedule sharpens VOXZOGO commercial risk despite BioMarin's 20% U.S. royalty. Ascendis Pharma (ASND; Buy) reported Week 52 data from seven treatment-naïve infants with achondroplasia (ACH), mean age 11.7 months, treated with once-weekly YUVIWEL (navepegritide) 100μg/kg/week: annualized growth velocity (AGV) was 9.9 cm/year, ACH-specific supine-length Z-score increased 0.42, AFMS was stable or improved in 7/7, mean sagittal foramen magnum diameter increased 3.15 mm, and no decompression surgeries occurred. The cohort is small and uncontrolled. We believe 52

BioMarin Pharmaceutical Inc. (BMRN) PT Raised to $90; Piper Sandler: 'Remain Buyers'

Tue, 01 Sep 2026 09:00:04 -0400

(Updated - September 1, 2026 9:00 AM EDT)

Piper Sandler analyst Allison M. Bratzel raised the price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to $90.00 (from $88.00) while maintaining a Overweight rating.

The analyst commented: "Yuviwel IP Settlement Removes Overhang, Adds Royalty Revenue; Remain OW rated on BMRN and raising PT on today's news of a settlement with ASND (Ascendis, not covered) over Yuviwel, the long-acting CNP analog for achondroplasia that competes with Voxzogo. The settlement resolves global patent disputes, including the ongoing US ITC (International Trade Commission) case, in exchange for 20% royalties on net sales of Yuviwel in the

BioMarin Pharmaceutical Inc. (BMRN) PT Raised to $90; Piper Sandler: 'Remain Buyers'

Tue, 01 Sep 2026 09:00:04 -0400

(Updated - September 1, 2026 9:00 AM EDT)

Piper Sandler analyst Allison M. Bratzel raised the price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to $90.00 (from $88.00) while maintaining a Overweight rating.

Canaccord Reiterates Buy Rating on BioMarin Pharmaceutical Inc. (BMRN)

Tue, 01 Sep 2026 06:58:02 -0400

Canaccord analyst Whitney Ijem reiterated a Buy rating and $114.00 price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN).

The analyst commented: "Overnight, BMRN announced a global settlement with Ascendis (not covered) resolving all pending patent disputes related to Yuviwel (navepegritide), Ascendis' once-weekly CNP for achondroplasia. Under the terms, Ascendis will pay BMRN a royalty of 20% on net sales in the U.S., retroactive to the first commercial sale, and 18% on net sales in the EU, Brazil, and South Korea through May 2030. The settlement includes a license to BMRN's patents covering Yuviwel for all current and potential indications, including achondroplasia

BioMarin Pharmaceutical Inc. (BMRN) PT Raised to $60 at H.C. Wainwright

Mon, 31 Aug 2026 11:02:24 -0400

H.C. Wainwright analyst Mitchell Kapoor raised the price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to $60.00 (from $59.00) while maintaining a Neutral rating.

Morgan Stanley on BioMarin Pharmaceutical Inc. (BMRN): 'we think the settlement recognizes the value of BioMarin’s pioneering CNP innovation'

Mon, 31 Aug 2026 11:01:49 -0400

Morgan Stanley analyst Sean Laaman reiterated an Overweight rating and $124.00 price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN).

The analyst comments "By resolving the dispute ahead of a final ITC outcome, BioMarin secures economic value now through retroactive and prospective royalties. The settlement resolves every pending proceeding between the two parties and grants Ascendis broad freedom to operate. BioMarin will dismiss the pending Section 337 investigation, and both sides will resolve all claims relating to the asserted IP. The license extends to BioMarin's Yuviwel-related patents across all current and potential indications and also covers use of Yuviwel in combination with

BioMarin Pharmaceutical Inc. (BMRN) PT Raised to $105 at Jefferies

Mon, 31 Aug 2026 11:00:49 -0400

Jefferies analyst Akash Tewari raised the price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to $105.00 (from $104.00) while maintaining a Buy rating.

The analyst comments "BMRN announced a global settlement w/ ASND for Yuviwel, which A) gives BMRN royalties on Yuviwel net sales through May 2030 of 20% in the US & 18% EU/BR/KR, and B) resolves all pending patent-related proceedings btwn the co's. We think the agreement adds ~$1/sh NPV and more importantly validates BMRN’s CNP IP + adds credibility to mgmt., removes the Vox litigation overhang, and allows BMRN to share in pot'l upside if Yuviwel gains share.