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Monday, November 14, 2022

FDA Approves Relyvrio (sodium phenylbutyrate/taurursodiol) for Patients with Amyotrophic Lateral Sclerosis (ALS)


In continuation of my update on phenyl butyrate and taurursodiol..
Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”)   announced  the U.S. Food and Drug Administration (FDA)   approval of Relyvrio (sodium phenylbutyrate and taurursodiol) for the treatment of adults with amyotrophic lateral sclerosis (ALS). Relyvrio (previously known as AMX0035 in the U.S.) significantly slowed the loss of physical function in people living with ALS in a randomized, placebo-controlled clinical trial. Relyvrio can be taken as a monotherapy or with existing approved treatments.

“Today’s FDA approval of Relyvrio is an exciting milestone for the ALS community and is a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. “We want to give a heartfelt thank you to the broader ALS community, including healthcare professionals and those living with ALS, for their guidance, support of our clinical programs, and for sharing their experiences with us. Their stories inspired us and helped our team to better understand the ALS clock, instilling in us a deep sense of urgency that will continue to drive us forward. This is just the beginning and there is much more to be done.”
ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis and eventually, death. More than 90% of people with ALS have sporadic disease, showing no clear family history. ALS affects approximately 29,000 people in the U.S.

Leading U.S. ALS advocacy organizations including The ALS Association, Answer ALS Foundation, I AM ALS, Les Turner ALS Foundation and Team Gleason said in a statement, “Our organizations have been on a mission to create a world free of ALS. With today’s approval, we are encouraged that Relyvrio can offer people living with ALS and their families the potential of more time with functional independence. This is especially important for a rapidly progressive disease with a median survival time from diagnosis of just two to three years. This is significant for people living with ALS, their loved ones, caregivers, clinicians, researchers, and advocacy, as we now have a new treatment option that could be a big step forward for the future of ALS care.”

The approval of Relyvrio is based on data from CENTAUR, a multicenter Phase 2 clinical trial in 137 participants with ALS encompassing a 6-month randomized, placebo-controlled phase and an open-label extension (OLE) long-term follow-up phase. Detailed data from CENTAUR were published in the New England Journal of MedicineMuscle & Nerve, and the Journal of Neurology, Neurosurgery, and Psychiatry.

The most common adverse events occurring with Relyvrio (at least 15% and at least 5% greater than placebo) were diarrhea, abdominal pain, nausea, and upper respiratory tract infection. Gastrointestinal-related adverse reactions occurred throughout the study but were more frequent during the first three weeks of treatment.

“Any time we have a new tool to slow the progression of this disease represents an important milestone in how we battle ALS. The published data on both function and survival in a randomized trial – and what this means for people living with ALS – are a step forward for the ALS community,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, and Associate Professor of Physical Medicine and Rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital.


FDA Approves Relyvrio (sodium phenylbutyrate/taurursodiol) for Patients with Amyotrophic Lateral Sclerosis (ALS)

Monday, December 27, 2021

Amylyx Pharmaceuticals Submits New Drug Application (NDA) for AMX0035 for the Treatment of ALS

 

Sodium phenylbutyrate

Sodium phenylbutyrate Structural Formula V1.svg          

 

 

Taurursodiol

 

 

 

Amylyx Pharmaceuticals, Inc.  announced that, it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for AMX0035 (sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine)) for the treatment of amyotrophic lateral sclerosis (ALS).

“We are excited to share with the ALS community the exciting milestone that we have submitted our NDA to the FDA for review,” said Justin Klee, Co-CEO, Director and Co-Founder of Amylyx. Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx added, “Our team has worked and continues to work around the clock as we know time is of the essence for people living with ALS and their families. We will continue to keep the community closely updated on our progress.”
“We will continue to work closely with the FDA throughout the review process to move AMX0035 toward a potential regulatory approval as quickly and efficiently as possible,” said Tammy Sarnelli, Global Head of Regulatory Affairs of Amylyx. “We are so inspired by the people who participated in CENTAUR, the trial investigators, the ALS community and our partners and team, and we will continue to work tirelessly on behalf of you all.”

The NDA submission to the FDA is based on data from the CENTAUR trial, a placebo-controlled study evaluating 137 people with ALS. In this study, participants receiving AMX0035 had statistically significant slowing of functional decline at the end of the 6-month randomized phase as measured by the Revised ALS Functional Rating Scale (ALSFRS-R), the most commonly used scale in clinics worldwide to measure function in ALS. In an interim survival analysis conducted in all randomized participants from CENTAUR who were followed for up to three years, which included participants who continued to receive AMX0035 in an open-label extension phase during the follow-up period, participants who started on AMX0035 during the placebo-controlled phase of CENTAUR showed a 44% lower risk of death compared to those who started on placebo during the placebo-controlled phase (HR 0.56; 95% CI, 0.34-0.92). Median survival duration through the open-label long-term follow-up phase was 25.0 months (95% CI, 19.0-33.6 months) in the group that started on AMX0035 and 18.5 months (95% CI, 13.5-23.2 months) in the group that started on placebo, a 6.5-month difference. Overall, reported rates of adverse events and discontinuations were substantially similar between AMX0035 and placebo groups during the 24-week randomized phase; however, gastrointestinal events occurred with greater frequency (≥2%) in the AMX0035 group. Detailed data from CENTAUR is published in the New England Journal of Medicine (NEJM) and Muscle and Nerve.

“This submission brings hope to people living with ALS and their families and caregivers,” said Merit Cudkowicz, M.D., co-principal investigator of the CENTAUR trial and co-founder of the Northeast ALS Consortium, Director of the Healey & AMG Center for ALS and Chair of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “We are honored to have led the collaboration between the Healey & AMG Center for ALS, ALS Finding a Cure, the ALS Association, and NEALS that made the CENTAUR trial a reality, the efforts and results of which made this NDA submission possible.”

“For people living with ALS and their physicians, this is a significant development offering hope of a potential new treatment option that has been shown to slow ALS disease progression and extend the time families that face this life-threatening disease have together,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital.

As previously reported, Amylyx filed a New Drug Submission (NDS) for AMX0035 for the treatment of ALS with Health Canada in June 2021. Amylyx also intends to submit a Marketing Authorization Application (MAA) for AMX0035 to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) by approximately the end of 2021 and to initiate a global Phase 3 clinical trial with sites in Europe and the United States in the fourth quarter of 2021. The Phase 3 PHOENIX trial of AMX0035 for the treatment of people with ALS will assess the safety and efficacy of AMX0035 in an international population of approximately 600 participants and build upon findings from the CENTAUR trial. Amylyx is currently exploring the possibility of an Expanded Access Program (EAP) in the United States. If implemented, the EAP would run in parallel with the ongoing Phase 3 PHOENIX trial and marketing application reviews. Further information about the EAP is expected in the fourth quarter of 2021.

 More

https://en.wikipedia.org/wiki/Sodium_phenylbutyrate

https://www.kegg.jp/entry/D11836