Rare diseases may seem niche, but their impact is far from small. An estimated 7,000 rare diseases exist, collectively affecting a 300 million people worldwide. This immense burden of disease, coupled with a profound lack of treatment options, underscores the urgent need for innovation. “Actually, the total burden of disease and unmet medical need [for…
Amylyx Pharmaceuticals launches phase 2 trial for AMX0035 Wolfram syndrome therapy
Cambridge, Massachusetts–based Amylyx has dosed the first participant in its phase 2 HELIOS clinical trial of AMX0035 focusing on Wolfram syndrome. The condition is an ultra-rare genetic disorder involving the endocrine system. Symptoms of Wolfram syndrome can include diabetes insipidus, diabetes mellitus, optic atrophy and hearing loss. In September, Amylyx won FDA approval for AMX0035…
Chiesi Farmaceutici acquires Amryt Pharma to bolster access to rare disease therapies
Chiesi Farmaceutici, a family-owned international pharma company, has completed its $1.25 billion acquisition of rare disease-focused biopharma Amryt Pharma (Nasdaq:AMYT). The companies announced the deal in January and projected it would close in the first half of 2023. Strengthening Chiesi’s global rare disease focus Chiesi’s decision to acquire Amryt Pharma is part of a broader…
Prominent pharma M&A deals to watch in Q1 2023: Pfizer, Sanofi and others on the hunt
After a relatively quiet 2022, 2023 is anticipated to be a more robust year for pharma M&A activity. In recent months, players such as Pfizer (NYSE:PFE), Sanofi (NYSE:SNY), Chiesi, AstraZeneca (LON:AZN) and Amgen (Nasdaq:AMGN) have unveiled acquisition plans that could reshape the landscape of the sector. This article delves into the details of prominent recent…
Pharmaceutical Companies Are Profiting from Rare Diseases
Incentives intended to stimulate the development of more treatments for rare diseases are being exploited to boost the profits of pharmaceutical companies, new research led by Bangor University shows. Company profits Researchers found that companies which market drugs for rare diseases (known as orphan drugs) are 5 times more profitable and have up to 15%…