- La tensión Madrid-Buenos Aires acaba en crisis diplomática abierta
- Filme sobre Trump trae la política al Festival de Cannes
- El rey de Arabia Saudita padece una infección pulmonar
- El beneficio anual de Ryanair crece un 34% gracias al aumento de pasajeros y precios
- Luis Abinader, el presidente dominicano de mano dura con Haití
- Sean "Diddy" Combs pide disculpas tras un video que lo muestra agrediendo a su expareja
- El papa Francisco califica de "locura" las actitudes antimigrantes en la frontera de EEUU
- Lai asume como presidente de Taiwán y pide el fin de la "intimidación" china
- El presidente dominicano es reelegido para un segundo mandato marcado por Haití
- El presidente iraní Raisi muere en un accidente de helicóptero
- Ebrahim Raisi, un presidente iraní ultraconservador
- Medios iraníes anuncian el deceso del presidente Raisi en accidente de helicóptero
- Lai asume como presidente de Taiwán y pide fin de "intimidación" de China
- España llama a consultas a su embajadora en Argentina y exige disculpas a Milei
- "Ninguna señal" de vida en helicóptero accidentado del presidente de Irán
- Presidente dominicano reelecto para un segundo mandato marcado por Haití
- Presidente dominicano lidera elecciones marcadas por la crisis de Haití
- Taiwán inviste a un presidente detestado por China
- Biden enfrenta protesta silenciosa por Gaza y promete a estudiantes trabajar por la paz en Oriente Medio
- Irán continúa su intensa búsqueda del presidente Raisi tras un accidente de helicóptero
- Kevin Costner vuelve al wéstern en Cannes, Serebrennikov a los fantasmas de Rusia
- El estreno de "Lula" de Oliver Stone en Cannes, un acto de adhesión al mandatario brasileño
- Al menos once muertos en bombardeos rusos en la región ucraniana de Járkov
- El barco que destruyó el puente de Baltimore será movilizado el lunes
- Manchester City hace historia, Arsenal vuelve a ser segundo
- Biden promete a estudiantes escuchar protestas por Gaza y trabajar por "paz duradera" en Oriente Medio
- Blue Origin lleva pasajeros al espacio, incluido el astronauta con más edad
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- El presidente de Irán, ilocalizable tras un "accidente" de helicóptero
- Irán emprende búsquedas para encontrar el helicóptero del presidente Raisi tras "un accidente"
- Los bombardeos continúan en Gaza, emisario de EEUU en Israel
- Blue Origin lleva al espacio a aventureros tras dos años de pausa
US company withdraws ALS drug after it fails in trial
Amylyx Pharmaceuticals announced Thursday it was withdrawing its approved treatment against the deadly neurodegenerative disease ALS after clinical data found no evidence the drug worked.
In a statement, the US company said it would discontinue its market authorizations for Relyvrio/Albrioza, using the brand names of the medicine in the US and Canadian markets.
"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 and other neurodegenerative diseases," said the company's co-CEOs Joshua Cohen and Justin Klee in a statement.
The company also said it was reducing its workforce "by approximately 70 percent" as it focused on another experimental drug for use against ALS, and on repurposing Relyvrio for other conditions. It added it would continue to make Relyvrio available for patients who wish to keep using the treatment, through a "free drug program."
The news follows data from a clinical trial of 664 ALS patients announced in March, which found no significant differences in outcomes between those on the treatment group and those who received a placebo.
It was a big blow for patients with amyotrophic lateral sclerosis, sometimes called Lou Gehrig's disease after the famous baseball player, which devastates nerve cells in the brain and spinal cord.
ALS affects about two people per 100,000 every year, causing progressive loss of motor and cognitive function. Most patients die within five years of their diagnosis.
Relyvrio's approval by the US Food and Drug Administration in 2022 was controversial and based on the results of a single trial that involved just 137 participants.
The FDA itself noted there was "residual uncertainty about the evidence of effectiveness" -- but "given the serious and life-threatening nature of ALS and the substantial unmet need, this level of uncertainty is acceptable in this instance and consideration of these results in the context of regulatory flexibility is appropriate."
- Patient groups backed approval -
Advocacy groups also mounted a major campaign sending a petition to the FDA with tens of thousands of signatures urging approval. Once it became available, Amylyx reportedly announced an eye-watering list price of $158,000 per year in the US, drawing criticism.
Patient groups in Europe watched with desperation at the bureaucratic delays.
When the European Union drug watchdog later announced it was rejecting Relyvrio, the decision was slammed as "an affront" by angry French patients, who say they "don't have time to wait." France later relented, offering conditional approval in November.
"We commend Amylyx for pulling Relyvrio off the market, while still ensuring that people living with ALS can access the drug if they believe it is helping them," said the US-based ALS association, which had lobbied for the drug's approval and funded its research.
"Safe and potentially effective treatments can be made accessible rapidly until further research can confirm their efficacy," it added.
For now, there remain only a handful of treatments available.
Riluzole, FDA approved in 1995, prolongs life approximately three months. Edaravone, FDA approved in 2017, has been found to slow disease progression and improve survival.
And in 2023, the regulatory body approved tofersen, a gene therapy treatment that targets those ALS cases that are caused by mutations in the SOD1 gene.
K.Thomson--BTB