Rare Disease - MarketMinute

BioMarin

BioMarin’s $4.8 Billion Amicus Acquisition: A Strategic Pivot to Rare Disease Dominance

In a move that has sent shockwaves through the biotechnology sector, BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced on December 19, 2025, that it has entered into a definitive agreement to acquire Amicus Therapeutics (Nasdaq: FOLD) for approximately $4.8 billion. The all-cash transaction, valued at $14.50 per share, represents

Capricor Therapeutics

Capricor Therapeutics (NASDAQ: CAPR) Soars 535% to Eight-Year High on Landmark Duchenne Muscular Dystrophy Treatment Results

December 3, 2025 – Capricor Therapeutics (NASDAQ: CAPR) has sent shockwaves through the biotech sector today, witnessing an astonishing 535% surge in its stock price, propelling the company to an eight-year high. The dramatic ascent follows the announcement of overwhelmingly positive top-line results from its pivotal Phase 3 HOPE-3 study for

Agios Pharmaceuticals

Agios Pharmaceuticals Plunges Over 50% as Sickle Cell Drug Delivers Mixed Phase 3 Results

Boston, MA – November 19, 2025 – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) experienced a dramatic and immediate market backlash, with its stock plummeting over 51.00% following the announcement of mixed topline results from its pivotal RISE UP Phase 3 trial for mitapivat in sickle cell disease (SCD). While the investigational therapy

Biotech

Omeros Rockets Skyward on $2.1 Billion Novo Nordisk Deal, Reshaping Biotech Landscape

In a dramatic turn of events that sent shockwaves through the biotechnology sector, Omeros Corporation (NASDAQ: OMER) witnessed its stock catapult by an astounding nearly 150% after announcing a colossal licensing agreement with pharmaceutical titan Novo Nordisk (CPH: NOVO B). The deal, potentially valued at up to $2.1 billion,

Zevra Therapeutics

Zevra Therapeutics' Stock Soars on Strong Performance and Strategic Wins: What It Means for Investors

Zevra Therapeutics (NASDAQ: ZVRA) has captured significant attention in the financial markets with its recent robust stock performance and a compelling "Strong" Relative Strength Rating. As of early October 2025, the biopharmaceutical company, focused on rare diseases, is demonstrating a positive trajectory, fueled by critical regulatory milestones, impressive

Applied Therapeutics

Applied Therapeutics Soars Over 100% on Breakthrough CMT-SORD Trial and Renewed Investor Confidence

New York, NY – September 26, 2025 – Applied Therapeutics, Inc. (NASDAQ: APLT) ignited the biotech sector today, witnessing an astonishing stock surge of 103.50%. The dramatic leap comes on the heels of pivotal advancements for its lead drug candidate, govorestat (AT-007), targeting Charcot-Marie-Tooth Type 1A with Sorbitol Dehydrogenase (CMT-SORD) deficiency,