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FDA’s Go-Ahead to Amylyx New Drug Application for ALS Drug
Amylyx Pharmaceuticals has announced its plan to submit a New Drug Application (NDA) to the U.S. FDA for its drug, AMX0035 (sodium phenylbutyrate (PB) and Taurursodiol (TURSO)), for the treatment of Amyotrophic lateral sclerosis (ALS).
A few months ago, the company had decided to file for a marketing application to the different regulatory agencies seeking approval on the basis of the Phase II CENTAUR trial, which has shown that the drug showed survival benefit and met its primary endpoints. However, the USFDA had requested for Phase III trial to give its recommendation.
In a recent announcement, the company made its intent to file for NDA clear that followed recent discussions with the FDA, including a pre-NDA meeting held on July 15, 2021.
Amyotrophic lateral sclerosis continues to pose a significant healthcare burden, with curative therapies appearing not less than evasive prey for the scientific community. The present ALS market comprises Radicava (Edaravone; Mitsubishi Tanabe Pharma), Rilutek (Riluzole; Covis Pharmaceuticals), Tiglutik (riluzole), Exservan and Nuedexta; however, none of the therapies are curative and are associated with concerns such as side effects or pricing.
However, the company is optimistic about its drug as they claim it to be the drug that managed to improve both function and survival.
FDA Approval to BeiGene’s Brukinsa for Advanced Marginal Zone Lymphoma
BeiGene is gushing with joy after the USFDA gave its nod to Brukinsa (zanubrutinib), a Bruton’s tyrosine kinase (BTK) inhibitor, for treatment for adult patients with relapsed or refractory (R/R) marginal zone lymphoma (MZL).
The approval is based on the findings from two clinical studies-phase 2 MAGNOLIA clinical trial and another phase 1/2 trial. The drug managed to shrink the disease with an impressive overall response rate.
Brukinsa ( Zanubrutinib) is a small-molecule inhibitor of Bruton tyrosine kinase (BTK) and is already approved for the treatment of mantle cell lymphoma patients who received at least one prior therapy. Recently the drug received approval for adults with Waldenström macroglobulinemia in the US and now managed to score another, expanding its profile.
Sage Therapeutics Witnesses a Rush in its Share Price
Sage Therapeutics shares leaped after the company announced that the USFDA had granted Fast Track Designation to its drug SAGE-718 for a debilitating disorder of the brain, Huntington’s disease (HD). The designation will help the company to take its orphan drug to patients in the market comparatively earlier.
Huntington’s disease is a rare, inherited neurodegenerative disorder with a prevalence of 80, 170 in 2020 in the 7MM, which is further expected to grow in the coming years. The rare disorders market faces a dearth of curative therapies despite widespread cumulative prevalence owing to several challenges such as difficulty in conducting trials, lack of understanding and knowledge of pathophysiology and so on.
However, measures by the regulatory authorities such as providing market exclusivities, waivers in fees and taxes and so on have helped facilitate the development of rare drugs.
Roche, Temedica to Launch ‘Digital Companion’ for Multiple Sclerosis
Germany’s Temedica and Swiss pharma giant Roche have come together in pursuit of providing digital support to Multiple sclerosis patients. The duo has decided to design a new mobile app to deepen understanding of the disease based on real-world data gained from Multiple Sclerosis patients.
The Brisa platform leverages Roche’s medical expertise compounded with Temedica’s experience in developing digital health tools.
The mobile app, dubbed Brisa, is expected to collect daily health data like activity, sleep, and dietary habits to help track the symptoms of Multiple Sclerosis, which will help the areas of improvement, suggest lifestyle changes to add to the efficiency of the existing therapies. The app also has a feature to provide individualized therapy support while simultaneously sharing the updates with the physicians.
As the emerging technologies continue to fascinate and transform healthcare, several pharma companies are now looking out to expand their expertise in the therapeutic areas by exploring AI and digital means. Looking at an increasing number of collaborations between pharma tycoons and digital healthcare service providers, it is quite transparent that the digital era in healthcare has finally started to gain the traction and importance it deserved.