Amylyx Pharmaceuticals Announces FDA Advisory Committee Supports Approval of AMX0035 for the Treatment of ALS
- FDA Advisory Committee voted 7:2 that the available evidence of effectiveness is sufficient to support approval of AMX0035 for the treatment of ALS
- If approved, AMX0035 will be the first treatment in ALS that has demonstrated a significant slowing of disease progression and functional decline, as well as extended survival, in a randomized, placebo-controlled clinical trial, as a standalone therapy or when added to existing approved treatments
The PCNSDAC’s decision was based on a review of all available evidence, including new analyses submitted for discussion at the
“The Committee’s thoughtful review of the data and support of the benefit that AMX0035 may bring to the ALS community, if approved, is promising,” said
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.
The PCNSDAC recommendations, while not binding, will be considered by the FDA in its review of the pending New Drug Application (NDA) for AMX0035. As previously reported, the Prescription Drug User Fee Act target action date for the NDA is
AMX0035 (sodium phenylbutyrate and taurursodiol) is an oral fixed-dose medication with marketing applications pending in the
About the CENTAUR Trial
CENTAUR was 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. The trial met its primary efficacy endpoint.
CENTAUR results appear to show that AMX0035 was generally well tolerated. Similar rates of adverse events and discontinuations were recorded in the AMX0035 and placebo groups during the 24-week randomized phase. However, gastrointestinal (GI) events occurred with greater frequency (≥2%) in the AMX0035 group. Detailed safety and functional efficacy data from CENTAUR were published in the
The CENTAUR trial was funded, in part, by the ALS ACT grant and the ALS Ice Bucket Challenge, and was supported by
The Phase 3 PHOENIX clinical trial (NCT05021536) is a 48-week, randomized placebo-controlled global clinical trial further evaluating the safety and efficacy of AMX0035 (sodium phenylbutyrate and taurursodiol) for the treatment of ALS. The primary efficacy outcome of the trial will be a composite measure of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score progression over 48 weeks and survival over 48 weeks. Secondary endpoints include change in slow vital capacity (SVC), measured both at home using a self-administered spirometer to support virtual data collection and at clinic sites using standard spirometry, quality of life patient-reported outcome assessments, ventilation-free survival rates and other measures.
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding beliefs about the clinical study analyses of AMX0035, the potential additional regulatory approvals of AMX0035 and the Company’s efforts to advance such potential approvals and the potential of AMX0035 or other future therapeutic candidates as a treatment for ALS, and expectations regarding our longer-term strategy. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’ program development activities, Amylyx’ ability to execute on its strategy, regulatory developments, expectations regarding the timing of regulatory review of AMX0035 and the inherent unpredictability of the regulatory review process, Amylyx’ ability to fund operations, and the impact that the ongoing COVID-19 pandemic will have on Amylyx’ operations, as well as those risks and uncertainties set forth in Amylyx’