Following the recent shift in the FDA’s stance on Amylyx Pharmaceuticals’ AMX0035* new drug application (NDA), leading data and analytics company, GlobalData, expects that this drug could be available for amyotrophic lateral sclerosis (ALS) patients much sooner than anticipated.

Sarah Elsayed, Neurology Analyst at GlobalData, comments: “As AMX0035 targets a serious neurological condition with huge clinical unmet needs, it could be granted an FDA accelerated approval within one year from the NDA filing – if the FDA follows a similar path to the controversial approval of Biogen’s Aduhelm in Alzheimer’s disease earlier this year.”

Based on the positive results of Phase II CENTAUR trial, AMX0035 has been filed for approval in Canada in June 2021 and is expected to be filed in the EU by the end of 2021.

Elsayed continues: “Key opinion leaders (KOLs) interviewed by GlobalData believe that AMX0035 has a favorable oral administration and a well-known safety and tolerability profile. They indicated that they would likely prescribe AMX0035 to the majority of their patients upon approval, if it continued to show therapeutic benefits in trials. This is because the drug addresses a number of clinical unmet needs and expands existing treatment options in the ALS market. However, they hoped that the drug will be priced reasonably, due to the availability of its two constituents.

“KOLs noted that, given the devastating nature of ALS and the average short life expectancy of patients, there is a strong public pressure to get more drugs approved for this disease – despite showing modest benefits on survival and slowing disease progression. As such, GlobalData expects AMX0035 to gain a slice of the ALS market, which is expected to grow at a compound annual growth rate (CAGR) of 13.9%, as well as reach $1.04bn in global sales by 2029, driven by pipeline launches**.”

“Overall, GlobalData anticipates that the need for a drug with curative and disease-modifying properties remains, however, new entrants in this stagnant market will likely encourage companies to invest in developing pipeline agents with novel mechanisms of action that try to halt or slow the progression of the disease, rather than reformulating available ALS treatments.”

* tauroursodeoxycholic acid and sodium phenylbutyrate

**Data taken from GlobalData’s report: GlobalData (2020) Amyotrophic Lateral Sclerosis (ALS): Opportunity Analysis and Forecasts to 2029