Metachromatic Leukodystrophy

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Bristol Myers Squibb’ Karuna Therapeutics Buyout; Orchard Therapeutics’ Lenmeldy FDA Approval; Madrigal Pharmaceuticals’ Rezdiffra FDA Approval; AstraZeneca’s Acquisition of Amolyt Pharma; Prilenia’s Pridopidine EU Marketing Approval

Bristol Myers Squibb Enhances Neuroscience Arm with Karuna Therapeutics Buyout Bristol Myers Squibb has declared the finalization of its purchase of Karuna Therapeutics, Inc. With this acquisition concluded Karuna’s shares are no longer being traded on the Nasdaq Global Select Market, as Karuna is now fully owne...

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Orchard’s OTL-200 to Enter US Metachromatic Leukodystrophy Treatment Space After EU

Orchard Therapeutics’ Biologics License Application for OTL-200, a gene therapy under investigation for metachromatic leukodystrophy treatment, has been accepted by the FDA. This rare disease treatment received approval in Europe back in 2020. Notably, the FDA has granted Orchard’s application Priority Review statu...

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Biogen’s Aduhelm; FDA Approves Sanofi’s Enjaymo; NHS & Orchard Signs a Deal; Bristol’s Breyanzi & Abecma; Pharming’s Leniolisib; Eli Lilly’s Verzenio Trial; UCB’ Zilucoplan Trials Result; Eli Lilly’s Alzheimer’s Drug Donanemab

Biogen's Aduhelm Marketing and Approval are Under Scrutiny in New FTC and SEC Investigations Biogen can't seem to get a break when it comes to the Alzheimer's disease drug Aduhelm. Along with limited sales and a constraining Medicare coverage plan, the medicine is now facing additional scrutiny as part of a pair...

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Metachromatic Leukodystrophy (MLD): A Rare Indication with great unmet medical need

Metachromatic Leukodystrophy (MLD) is a rare lysosomal storage disease which is genetic, degenerative, neurometabolic in nature. It is generally inherited from carrier parents and at present doesn’t have any cure. Patients suffering from MLD are deficient in the arylsulfatase-A enzyme, which is responsible for break...

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