United States – The maker of the Lou Gehrig’s disease drug who recently failed the clinical study will take the medication off the market by Thursday. His decision came after he detected that the condition did not improve the patients’ lives at all.
Amylyx Pharmaceuticals Halts Sales
Amylyx Pharmaceuticals made a voluntary decision to stop sales and marketing of the drug of a new, improved version in the U.S. and Canada, and new patients cannot get a prescription anymore, as reported by HealthDay.
“While this is a difficult moment for the ALS community, we reached this path forward in partnership with the stakeholders who will be impacted and in line with our steadfast commitment to people living with ALS,” co-founders said in a statement. The patients currently having this therapy can be enrolled in a program designed to access the drug without paying any money.
Regulatory Implications and FDA Approval
The U.S. Food and Drug Administration authorized the controversial drug, Relyvrio, in September 2022, after years of patient advocacy by ALS sufferers and their families.
The drug’s failure is an exceptionally bitter reality for patients and advocates who have been pushing hard for the FDA and other public agencies to allocate more resources and bring more clinical trials for this disastrous muscular dystrophy.
Only one of the only three ALS medicines available in the USA has been shown to prolong the disease over several months.
Amylyx, based in Cambridge, Massachusetts, commented that a new phase of reorganization would affect 70% of its 350 employees. According to the company directors, their next plans are to review Relyvrio and another experimental drug for rare diseases, including Wolfram syndrome, which causes diabetes and blindness from childhood.
By early morning on Thursday, company shares had already jumped almost 7.5%.
Amylyx mentioned slightly earlier that it was going to suspend its drug administration in the trial involving 600 patients because of the negative results, such as no improvements in the patient’s survival time, muscle strength, and walking ability.
Since the company took a voluntary action, a major issue for the FDA could have been raised. As regulator, the agency would not have a straightforward way of getting the drug off the market even if the manufacturer resisted. This is because the FDA approved the drug on a full basis instead of posterior data on its efficiency.
The 2022 approval, it should be mentioned, was based largely on the results of a single small, mid-stage study, which was criticized by some of the agency’s internal scientists. Usually, the agency needs two expensive, late-stage trials, which clearly show better outcomes, before this privilege is given. But then the officials of the FDA said that “regulatory flexibility” was suitable when reviewing Relyvrio, under the consideration of the fact that “given the serious and life-threatening nature of ALS and the substantial unmet need.”
This drug is one of the drugs for lethal diseases that have been approved by the FDA in recent years, but it is almost impossible to establish whether there is evidence to show they produce the desired effects.
ALS proceeds with the gradual obliteration of the nerve cells and connections needed to walk, speak, talk, and even breathe. The majority of patients find themselves getting close to death within less than three to five years after being diagnosed.
Patient Response to Relyvrio
Relyvrio is a powder that combines two older drugs: The examples are hepatitis drugs and a traditional Chinese medicine supplement, as reported by HealthDay.
Amylyx has been criticized for pricing the drug at USD 158,000/year in a blindfolded way. The sales were not satisfactory, with some patients quitting the drug right after only a few months.