April 22nd 2024
Prademagene zamikeracel is a cell therapy designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells. The FDA is asking for additional information on manufacturing practices.
April 11th 2024
FDA Issues Complete Response Letter for Onpattro in Heart Failure Indication
October 9th 2023Alnylam Pharmaceuticals will no longer pursue this indication of Onpattro and will instead on focus on a label expansion for Amvuttra, which is in phase 3 development to treat patients with cardiomyopathy of ATTR amyloidosis.
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Oncology Drug Exkivity to be Withdrawn from U.S. Market
October 3rd 2023In a confirmatory trial, Exkivity did not meet the primary endpoint in treating patients with non-small cell lung cancer with EGFR exon 20 mutations. It will remain available while Takeda works with the FDA on withdrawal timing.
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Inflation Reduction Act: Reforms to Patient Cost-Sharing
September 18th 2023Lower out-of-pocket costs for patients might put upward pressure on drug prices, as manufacturers face less price sensitivity, note Matthew Majewski and Rhett Johnson of Charles River Associates. But they also note that upward pressure on price is likely to be limited to the inflation rate as any additional price increase would need to be paid back to CMS in the form of inflation rebates.
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FDA Advisory Committee Backs Use of Onpattro in Heart Failure Indication
September 16th 2023Even though committee members voted in support of Onpattro for patients with cardiomyopathy related to transthyretin-mediated amyloidosis, there were questions about whether it provided a clinically meaningful benefit. The FDA set an action date of Oct. 8, 2023.
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FDA Approves Aphexda for Use in Stem Cell Mobilization
September 12th 2023In clinical trials, Aphexda plus filgrastim enabled a majority of patients to achieve the collection goal to enable stem cell transplantation for patients with multiple myeloma. It will have a list price 5,900 per vial, with most patients needing two vials for treatment.
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X4 Pharmaceuticals Submits NDA of Mavorixafor for Rare Immunodeficiency Disease
September 5th 2023If approved, mavorixafor would be the first therapy to address the genetic defect that results in WHIM syndrome, an ultra-rare disease that can cause recurrent lung infections, papillomavirus-related warts, and an increased risk of developing certain types of cancer.
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