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BioMarin Pharmaceutical

BioMarin Pharmaceutical Anonymous (not verified) Mon, 06/22/2026 - 00:00 field_thread_url https://cafepharma.com/boards/threads/biomarin-pharmaceutical.710...

BioMarin Pharmaceutical

BioMarin Pharmaceutical Anonymous (not verified) Fri, 06/12/2026 - 17:51 field_thread_url https://cafepharma.com/boards/threads/biomarin-pharmaceutical.710...

BioMarin notches win in study that could expand use of top-selling medicine

The success came two days after a Phase 3 setback for another experimental drug and helps improve the position of Voxzogo amid increased competition.

BioMarin drug acquired in buyout misses goal in rare disease study

Tested in a condition known as ENPP1 deficiency, the treatment failed a trial goal that’s important to regulators, spurring doubts about its approval prospects. 

BioMarin’s Mixed Bag of Phase 3 Data Dim Prospects for Rare Disease Drug

A BioMarin Pharmaceutical drug met just one of two main goals of a Phase 3 test in a rare inherited enzyme deficiency with no FDA-approved therapies. The drug, a fusion protein, ca...

Biomarin Pharmaceutical (BMRN): Most Undervalued Stocks to Buy and Hold for 2 Years

Novartis RNA drug acquired in $12B Avidity deal notches a trial win

The data showed that del-brax, an “antisense oligonucleotide conjugate” being tested against a muscle-wasting disease, met its primary endpoint in a Phase 1/2 study.

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J&J looks to widen Imaavy’s use; $300M backs rare disease drug launch

New data suggest Imaavy could be an effective treatment for autoimmune anemia. Elsewhere, Enliven detailed data for a Merck leukemia rival and MBX hyperparathyroidism drug hit its...

<![CDATA[Parabilis Medicines Raises $670 Million in Initial Public Offering ]]>

Novartis RNA Med From $12B Deal Keeps Building Case to Become First Therapy for Rare Muscle Disease

Novartis said its RNA therapy del-brax met goals of a Phase 1/2 study in facioscapulohumeral muscular dystrophy (FSHD), an inherited muscle disorder that currently has no FDA-appro...

Sanofi's Nexviazyme hits primary goal in Pompe disease study

Novartis' phase 1/2 win tees up regulatory talks about dystrophy drug from $12B Avidity takeover

Novartis has reported a phase 1/2 win for one of the jewels of its $12 billion Avidity Biosciences buyout, setting the Swiss drugmaker up to talk to global regulatory agencies abou...

Rocket Pharma Reaps $180M From Sale of FDA Drug Review Fast Pass

Rocket Pharmaceuticals received the rare pediatric disease priority review voucher with the accelerated FDA approval for Kresladi in March. The non-dilutive capital from the vouche...

Mirum Pharmaceuticals: This Liver Disease Juggernaut Just Broadened Its Portfolio

Ipsen’s $450M blood cancer drug; FDA selects first cohort for “pre-check” pilot program

The planned acquisition of Kartos Therapeutics would hand Ipsen a late-stage asset for myelofibrosis. Elsewhere, Sobi hit a regulatory setback and BeOne unveiled more positive Bruk...

FDA extends Beren’s rare disease review after info request, further stretching saga

The FDA has delayed its approval decision on Beren Therapeutics’ rare disease drug candidate, extending the asset’s long path toward the U.S. market.

Servier buys into muscular dystrophy with potentially $2.7B deal

The French drugmaker has agreed to acquire an experimental tablet from Edgewise Therapeutics that some bullish analysts say could eventually hit peak sales of over $2 billion.

Sangamo holds a bankruptcy sale; VCs back a China-linked Beam competitor

Eli Lilly and Astellas will be the lead bidders at an auction for Sangamo assets. Elsewhere, an investor syndicate committed $230 million to a gene editing startup and Merck KGaA s...

<![CDATA[Pharmaceutical Executive Daily: Parabilis Medicines Prices Record-Breaking $670 Million IPO]]>

City banks nearly $100M for next-gen RNAi drugs

Co-founded by longtime Alnylam CEO John Maraganore, City has brought a clotting disorder drug into early human testing. An experimental therapy for Stargardt disease could soon fol...

<![CDATA[Novartis Announces $105 Million Collaborative Agreement with Antares Therapeutics]]>

<![CDATA[Ascidian and Eli Lilly Enter $1.9 Billion Global Research Collaboration to Develop RNA Exon Editors]]>