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CHMP meeting highlights – June 2024
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended several new medicines, including positive opinions for treatments for rare disorders.
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amyotrophic lateral sclerosis (ALS)
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Drug approval roundup – May/June 2024
This article highlights some of the key regulatory approvals granted in May and June in the EU and US so far this year, including for oligonucleotide and biologic therapies.
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Oligonucleotide ALS therapy approved in EU
The European Commission (EC) has granted Biogen its third rare disease treatment approval in the European Union.
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CHMP meeting highlights: February 2024
Ten new medicines, including a new oligonucleotide therapy for ALS, were recommended for approval at the Committee for Medicinal Products for Human Use (CHMP)’s recent meeting.
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Converging knowledge and technology to transform neuromuscular disease treatment
With the emergence of innovative therapeutic technologies and progress in the genetic understanding of diseases, Biogen’s Toby Ferguson explains why it is a promising time for the development of therapies for neuromuscular conditions such as amyotrophic lateral sclerosis (ALS).
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EMA accepts marketing application for ALS treatment
The EMA has accepted the marketing authorisation application for tofersen, which could be the first treatment for a rare genetic cause of ALS.
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$38 million will fund rare disease research over four-year period
Research for rare diseases including ALS will be supported over the next four years by $38 million in FDA funding.
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Sanofi to sell Japanese manufacturing plant
Sanofi’s Japan-based manufacturing site near Tokyo will be sold to a European contract development and manufacturing organisation.
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FDA and NIH partner to understand ALS
The Critical Path for Rare Neurodegenerative Diseases (CP-RND) partnership was launched to develop breakthrough treatments for neurodegenerative diseases like amyotrophic lateral sclerosis (ALS).
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AstroRx® shows promise in early phase amyotrophic lateral sclerosis trial
Amyotrophic lateral sclerosis (ALS) patients treated with AstroRx® had a 45 percent reduction in their disease progression rate.
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Promising interim results from ALS study testing IPL344
The data suggests disease deterioration was halved in the six Amyotrophic Lateral Sclerosis (ALS) patients treated with IPL344 over eight months.
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Experimental motor neurone disease therapy shows promise
Tofersen reduced expression of the SOD1 gene known to cause amyotrophic lateral sclerosis (ALS) and slowed clinical decline in the Phase I/II trial.
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The FDA has approved Exservan™ for the treatment of ALS
The FDA has granted early-action approval to Exservan for the treatment of ALS, which can be administered safely and easily without water.
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Complete response letter given to ALS treatment for API concern
The FDA has handed a CRL to Biohaven for its ALS treatment awaiting approval due to concerns regarding the medication’s API.
