The recently launched phase 2 HELIOS clinical trial from Amylyx Pharmaceuticals, aims to explore the potential of AMX0035 (sodium phenylbutyrate and taurursodiol) in treating Wolfram syndrome, a rare and complex genetic disorder. In a recent interview, Drs. Lahar Mehta and Fumihiko Urano discussed the AMX0035 Wolfram syndrome trial design, objectives and implications for future research…
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…
FDA approves Amylyx ALS drug Relyvrio
Amylyx Pharmaceuticals (Nasdaq:AML) has won FDA approval for Relyvrio (AMX0035; sodium phenylbutyrate and ursodoxicoltaurine), notching the first win for amyotrophic lateral sclerosis (ALS) in five years. The drug received Health Canada approval in June. The drug is known as Albrioza in that country. In a summary, FDA reviewers note that the limited clinical data available…
Inside Canada’s approval of Amylyx’s ALS drug Albrioza
One of the things that differentiates amyotrophic lateral sclerosis (ALS) from other rare diseases is that “it is a catastrophic diagnosis,” said Chris Aiello, Head of Canada and General Manager, Amylyx Pharmaceuticals. “Patients do not really have much to go on in terms of pharmacological treatments.” Last week, Amylyx Pharmaceuticals received Health Canada approval for…
Understanding the promise of Albrioza: Q&A with the CEO of ALS Society of Canada
Amyotrophic lateral sclerosis (ALS) remains a bewildering disease. Although it is rare, it is not uncommon. The lifetime risk of developing the condition is roughly 1 in 300 by age 85. The heterogeneity of the disease is also unique, said Tammy Pighin Moore, CEO of the ALS Society of Canada. “About 5% to 10% of the people diagnosed…
Amylyx notches first global approval for oral ALS therapy Albrioza
Amylyx Pharmaceuticals (Nasdaq:AML) has received Health Canada approval for Albrioza (AMX0035). The FDA is anticipated to decide on Albrioza, which consists of sodium phenylbutyrate and ursodoxicoltaurine, by September 29, 2022. Amylyx is the first new therapy for ALS to win approval in Canada since 2018. The approval also is the first global regulatory approval for…
Amylyx Pharmaceuticals preps NDA filing for ALS drug AMX0035
Cambridge, Massachusetts–based Amylyx Pharmaceuticals intends to submit a New Drug Application (NDA) to FDA for AMX0035 (sodium phenylbutyrate and taurursodiol) to treat amyotrophic lateral sclerosis (ALS), which is also known as Lou Gehrig’s disease. The company decided to file after a series of meetings with the FDA, including a pre-NDA meeting on July 15. AMX0035…
Amylyx Pharmaceuticals wins orphan drug designation from FDA
The neurodegenerative-diseased-focused biotech Amylyx Pharmaceuticals announced that it had received orphan drug designation for AMX0035, a proprietary blend of sodium phenylbutyrate and tauroursodeoxycholic acid. The Cambridge, Mass.-based company won that designation to treat Wolfram syndrome, a rare genetic disease associated with diabetes, progressive optic atrophy, hearing loss and severe neurological disabilities. The prognosis for patients…