Japan Approves BridgeBio Pharma-AstraZeneca Partnered Rare Heart Disease Drug
Based on the terms of the agreement, BridgeBio will receive a $30 million milestone payment upon approval in Japan, as well ...
Alexion’s ravulizumab granted FDA orphan designation
Alexion Pharmaceuticals’ ravulizumab for the prevention of delayed graft function after renal transplantation was granted FDA orphan ...
The Pharma Letter17h
BridgeBio’s Beyonttra approved in Japan
Genetic diseases company BridgeBio Pharma has announced that the Japanese Ministry of Health, Labor and Welfare has approved ...
Beyonttra™ (acoramidis), the First Near-complete TTR Stabilizer (≥90%), Approved in Japan to Treat ATTR-CM
In the Japanese Phase 3 study, 0% mortality was reported over the 30-month treatment period and acoramidis was well-tolerated ...
The American Journal of Managed Care12d
FDA Approves Expanded Indication of Eculizumab for Pediatric Generalized Myasthenia Gravis
The FDA first approved eculizumab for use in adult patients with generalized myasthenia gravis in 2017, before expanding the ...
pharmaphorum3d
Alexion finally has a buyer – and it's AstraZeneca with $39bn on the table
Alexion has been working hard to flesh out is pipeline as well, snapping up Achillion Pharma, Syntimmune, Wilson Therapeutics and Portola and forging an alliance with gene-silencing specialist ...
pharmaphorum3d
Big pharma vs small diseases: Tackling rare diseases
head of Alexion’s new development hub, about these new ventures and about why and how big pharma tackles small diseases. Novo’s name change Novo Nordisk is far from new to the world of rare di ...
BridgeBio announces Japanese MHLW approved acoramidis for treating ATTR-CM
BridgeBio Pharma (BBIO) announced the Japanese Ministry of Health, Labour and Welfare, MHLW, has approved acoramidis, under the brand name ...
GlobalData on MSN9d
AstraZeneca’s $1bn hypoparathyroidism drug achieves success at Phase III
UK-based AstraZeneca’s acquired rare disease drug has met the primary endpoint in a Phase III trial in patients with chronic ...
FiercePharma27d
Arrowhead, Zevra fire up campaigns for Rare Disease Day 2025
Rare Disease Day 2025 triggered a flurry of pharma marketing activity ... AstraZeneca’s rare disease unit, Alexion, and Harmony Biosciences joined an episode of the morning show “The Balancing ...
Covington's London Office Secures $1 Billion AstraZeneca Mandate
The FTSE 100 company, which has a market capitalisation of £185.9 billion, confirmed the deal in an announcement to the ...