Search results for Biomarin Pharmaceutical

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 outlines $3.825B-$3.925B 2026 revenue outlook driven by Amicus integration and 20% growth target

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...

FDA Accepts Ultragenyx (RARE) BLA for Sanfilippo Syndrome Gene Therapy UX111

FDA Accepts Ultragenyx (RARE) BLA for Sanfilippo Syndrome Gene Therapy UX111

BioMarin’s $270M Inozyme bet looks in trouble after rare disease drug misses key ph. 3 goal

BioMarin’s $270 million bet on Inozyme’s enzyme replacement therapy looks in trouble after the candidate only managed to hit one of the dual primary endpoints of a phase 3 study in...

<![CDATA[Shionogi Enters $2.5 Billion Agreement to Acquire All Rights to Radicava]]>

Pfizer deals extend patent life for a top-selling rare disease drug

Settlements delaying the arrival of generic versions of tafamidis will protect billions in Pfizer revenue and have implications for others, including BridgeBio Pharma and Alnylam. 

<![CDATA[Angelini Pharma Enters $ 4.1 Billion Agreement to Acquire Catalyst Pharmaceuticals ]]>

Ultragenyx Pharmaceutical: The Market Is Overlooking 2026 Catalysts

<![CDATA[Biogen Enters $5.6 Billion Agreement to Acquire Apellis Pharmaceuticals ]]>

With new data, Regenxbio to seek FDA approval of Duchenne gene therapy

Though two serious side effects muddied the results, CEO Curran Simpson expressed optimism about a clearance and claimed FDA leadership will have a “mandate on rare disease flexibi...

<![CDATA[Pharma M&A Roundup: Bayer to Acquire Perfuse Therapeutics, Madrigal Enters Licensing Agreement for ARO-P...

<![CDATA[Rocket Pharmaceuticals Enters $180 Definitive Agreement to Sell its Priority Review Voucher ]]>

Regenxbio: 'Hold' On RGX-121 CRL MPS II And RGX-202 DMD Results

Efzimfotase alfa demonstrated positive results from global Phase III clinical programme in hypophosphatasia

This announcement contains inside information CHESTNUT randomised, open-label, active-controlled trial demonstrated safety and tolerability of efzimfotase alfa in paediatric patie...

<![CDATA[Haisco Enters $745 Million Exclusive License Agreement with AbbVie ]]>

Chiesi to buy KalVista in $1.9B deal for rare disease drug

The acquisition is the fifth of a biotech company so far this week and, according to some Wall Street analysts, proves pharma’s interest in new and emerging oral therapies for here...

<![CDATA[Pharmaceutical Executive Daily: Rocket Pharmaceuticals Sells Pediatric Disease Priority Review Voucher]]>

Angelini to buy Catalyst in $4B play for rare neuro drugs

The acquisition hands the Italian pharma a group of medicines approved to treat various CNS disorders and gives it wider market access in the U.S.

<![CDATA[Neurocrine Enters $2.9 Billion Agreement to Acquire Soleno Therapeutics ]]>

<![CDATA[UCB Enters $2.2 Billion Agreement to Acquire Candid Therapeutics ]]>

Mirum Pharmaceuticals prices $600M senior notes