International Medical Breakthrough | The world's first gene therapy for ALS has been approved, significantly delaying the progression of the disease

Clinical Breakthrough

The phase III trial showed that the rate of motor function decline in the treatment group decreased by 52%

The biomarkers of nerve filament light chains (NfL) in cerebrospinal fluid decreased by 67%

Some patients in the early stage show a reversal of symptoms

Therapeutic mechanism:
The intrathecal injection of ASO (antisense oligonucleotide) technology was adopted to precisely silence the expression of pathogenic genes. The treatment requires lumbar puncture and drug administration once every four months.

Patient witness:
"After the treatment, I was finally able to lift a coffee cup by myself," said Michael R. (43), a Boston patient who participated in the trial. "This is the first time in five years that I have felt the condition stop deteriorating."

Market Dynamics:

The price is set at $325,000 per year, which is lower than analysts' expectations

Novartis has committed to providing humanitarian aid programs for low-income countries

The EMA of the European Union is expected to complete the approval in the third quarter

Expert Comment:
"This is a milestone in the treatment of neurodegenerative diseases," emphasized Dr. Sabrina Paganoni, director of the ALS program at Harvard Medical School. "Although it is currently only applicable to 3% of ALS patients, it paves the way for the treatment of other genetic subtypes."

Industry impact

Shares of biotech company Ionis rose 11% after the market closed

The ALS Association announced an additional 100 million US dollars in research funds

The National Medical Products Administration of China has included it in the priority review

Extended Reading
There are approximately 500,000 ALS patients worldwide. Currently, only 5% of the cases have identifiable pathogenic genes. Scientists are exploring therapies against other gene mutations such as C9orf72 and FUS.

(Source: FDA Announcement, New England Journal of Medicine, Reuters Healthcare)