Here is information on the latest US FDA approvals, the week of October 9 – October 13, 2023
Velsipity (Etrasimod) for UC
The FDA has approved Velsipity (etrasimod) for treating moderate to severe active ulcerative colitis (UC) in adults. Etrasimod is an oral medication that binds to the sphingosine-1-phosphate (S1P) receptors, with strong binding affinity for receptors 1, 4, and 5. The approval of etrasimod, manufactured by Pfizer, was based on safety and efficacy data from the ELEVATE UC 52 trial, and the ELEVATE UC 12 trial, both published in The Lancet in March, 2023. Etrasimod is the second agent in the S1P class approved for ulcerative colitis in the United States. The other agent in this class ozanimod (Zeposia), manufactured by Bristol Myers Squibb, received FDA approval in May 2021.
Braftovi (encorafenib) and Mektovi (binimetinib) for NSCLC
The FDA has approved the combination of encorafenib and binimetinib in BRAF V600E–mutant, metastatic non–small cell lung cancer as detected by an FDA-approved test. The approval, granted to Array BioPharma (a Pfizer subsidiary), is backed by data from the phase 2 PHAROS study. Among the 59 treatment-naive patients in PHAROS, researchers reported an ORR of 75%. Of the patients, 59% had responses lasting at least 12 months. The Braftovi/Mektovi combination is already approved for use in patients with unresectable or metastatic melanoma with a BRAF mutation. In NSCLC, the V600E and V600K BRAF mutations occur in approximately 2% and 10% of NSCLC cases, respectively.
https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/210496s014lbl.pdf
Edison System for Non-Invasive Destruction of Liver Tumors
The FDA has authorized marketing of the HistoSonics Inc. Edison System for the non-invasive destruction of liver tumors, including unresectable liver tumors, using a non-thermal, mechanical process of focused ultrasound. This type of ultrasound generates cavitation, a process in which the static pressure of liquid is reduced below the liquid’s vapor pressure, creating small, vapor-filled cavities without the use of heat. This method differs from high-intensity focused ultrasounds which uses heat to destroy tissue and avoids cavitation. Histotripsy uses focused sound energy to produce controlled acoustic cavitation that mechanically destroys and liquifies targeted liver tissue, including tumors, at sub-cellular levels. Non-thermal focused ultrasound should be considered only in patients with a sufficient amount of functional liver reserve to withstand the destruction of the planned volume of liver tissue. According to HistoSonics, Inc., marketing authorization makes Edison the first and only histotripsy platform available in the Unites States.
https://histosonics.com/news/fda-awards-histosonics-clearance-of-its-first-of-a-kind-edison-histotripsy-system-2/
Complete Response Letter for Patisiran ATTR amyloidosis
The FDA declined to approve patisiran for ATTR amyloidosis despite a favorable recommendation from its advisers. In a complete response letter, issued to the manufacturer Alnylam Pharmaceuticals the FDA noted that patisiran did not meaningfully improve the condition of patients with heart muscle issues or cardiomyopathy caused by ATTR (transthyretin) amyloidosis, which is characterized by abnormal deposits of transthyretin proteins in organs and tissues. Patisiran, which has the brand name Onpattro, is already approved in the U.S. to treat nerve damage in adult patients with hereditary ATTR amyloidosis. The FDA’s decision would not impact Onpattro’s commercial availability, according to Alnylam. The rejection was a rare instance of the regulator going against its advisory panel’s backing for a drug. The FDA’s outside panel of experts had previously backed patisiran’s expanded use (as noted in the September 17, 2023 podcast episode), but the committee also raised concerns about the meaningfulness of its benefits. No issues were noted with the drug’s safety, quality, study conduct, or manufacturing, the company said. Alnylam said it will focus on another drug candidate, vutrisiran, an under-the-skin injection in late-stage trials to treat ATTR amyloidosis-related cardiomyopathy.
https://investors.alnylam.com/press-release?id=27716
FDA Creates a Digital Health Advisory Committee
The FDA has announced the creation of a new Digital Health Advisory Committee to help the agency explore the complex, scientific and technical issues related to digital health technologies (DHTs), such as artificial intelligence/machine learning (AI/ML), augmented reality, virtual reality, digital therapeutics, wearables, remote patient monitoring and software. The committee will consist of a core of nine voting members including the chair. The number of temporary members selected for a particular meeting will depend on the meeting topic. According to a written release from the FDA, The Digital Health Advisory Committee will provide “relevant expertise and perspective to help improve the agency’s understanding of the benefits, risks, and clinical outcomes associated with use of DHTs. The committee should be fully operational in 2024.”
https://www.accessdata.fda.gov/scripts/FACTRSPortal/FACTRS/index.cfm
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