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…
U.S. government launches CP-RND, a public-private partnership for rare neurodegenerative diseases
The FDA and NIH have debuted the Critical Path for Rare Neurodegenerative Diseases (CP-RND), a public-private partnership focused on developing new therapies for amyotrophic lateral sclerosis (ALS) and rare neurodegenerative diseases. One of the goals of the CP-RND is to foster innovation in drug discovery and development as well as clinical testing. In addition, CP-RND…
FDA launches 5-year initiative for rare neurodegenerative diseases
FDA has unveiled its Action Plan for Rare Neurodegenerative Diseases, including amyotrophic lateral sclerosis (ALS). The plan will involve the use of public-private partnerships and incorporate feedback from patients. The initiative will encompass regulatory science initiatives, changes to existing programs and new policy initiatives. Milestones of the initiative include the creation of an FDA rare neurodegenerative…
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…
Investigational ALS drug could slow disease progression
Irvine, Calif.-based Eledon Pharmaceuticals has announced promising topline results from a Phase 2a study of tegoprubart, marking the first time an investigational drug has demonstrated a reduction of inflammatory biomarkers associated with progression of amyotrophic lateral sclerosis (ALS). “We have an investigational drug acting based on what we hypothesized it would do based on the…
Scientists find new potential drug targets for neurodegenerative diseases
Researchers at Oregon State University (OSU) have discovered a new class of potential drug targets for diseases like Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS). The scientists are working to identify the best method to attack the targets — oxidized proteins. The most potent oxidant of the bunch is peroxynitrite, which is produced in conditions involving inflammation.…
QurAlis bolsters its executive team with three new leaders
Cambridge, Massachusetts–based biotech QurAlis Corp., has appointed Bryan Boggs as head of regulatory affairs. In addition, Christopher Gerry Lohan will join as head of clinical operations, and Guzide Adhikari will take on the role of head of global supply chain management. The company is focused on developing novel treatments for amyotrophic lateral sclerosis (ALS) and…
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…
QurAlis selects development candidate for ALS
Privately-held biotech QurAlis plans to begin clinical development for the first-in-class molecule QRL-201 for amyotrophic lateral sclerosis (ALS) in the second half of 2022. QRL-201 is now the subject of IND-enabling studies. The drug candidate targets the restoration of STATHMIN-2 (STMN2) expression in ALS. STMN2 is a protein encoded by the STMN2 gene that is involved in…
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…