In the Japanese Phase 3 study, 0% mortality was reported over the 30-month treatment period and acoramidis was well-tolerated ...

Based on the terms of the agreement, BridgeBio will receive a $30 million milestone payment upon approval in Japan, as well ...

BridgeBio Pharma (BBIO) announced the Japanese Ministry of Health, Labour and Welfare, MHLW, has approved acoramidis, under the brand name ...

UK-based AstraZeneca’s acquired rare disease drug has met the primary endpoint in a Phase III trial in patients with chronic ...

A phase 3 trial of AstraZeneca’s $800 million rare disease candidate has met its primary endpoint. The update keeps ...

In the Phase III CALYPSO trial, eneboparatide demonstrated statistical significance in achieving albumin-adjusted serum calcium normalization while eliminating the need for active vitamin D and oral ...

2-4 Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease, said: “People living with HypoPT, a rare endocrine disease, are often at increased risk of hypercalciuria, osteopenia and ...

Alexion, AstraZeneca Rare Disease will be responsible for all commercial activity for Beyonttra in Japan. “There is significant need for new treatment options for ATTR-CM, a progressive ...

AstraZeneca added eneboparatide to the Alexion and AstraZeneca rare disease pipeline through the acquisition of Amolyt Pharma in July 2024, in a deal valued around $1.05bn. Shareholders received ...

The approval in Japan is based on positive results from a Phase 3 open-label, single-arm study conducted in Japan by Alexion, AstraZeneca Rare Disease, and the positive results from the global ...