NHS Rolls Out £1.7 Million Gene Therapy for Sickle Cell Disease
The one-time gene-editing therapy Casgevy, developed by Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG, has a list price of £1.7 million ($2.1 million), but England's drug cost regulator said it has negotiated a lower, undisclosed price.
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World-first gonorrhea vaccine being rolled out in England
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The Hill
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Vertex next-generation pain candidate fails Phase 2 trial
'We do not plan to advance VX-993 as monotherapy in acute pain, because we do not expect that it will be superior to our [existing] NaV1.8 inhibitors,' said CEO Reshma Kewalramani during a Monday afternoon earnings call with investors, using a scientific shorthand for the class of drugs. She noted that the company will continue a trial testing the drug in patients with diabetes who have chronic nerve pain. It was one of multiple setbacks the company disclosed for its pain franchise Monday, as it announced its second-quarter earnings. Advertisement Vertex also announced that longtime chief scientist David Altshuler was retiring next year, to be replaced by head of global research Mark Bunnage. Altshuler, an accomplished academic geneticist, took the post in 2015 and presided over the launch of its most important Advertisement Although Journavx was On Monday, though, the company disclosed that the the Food and Drug Administration 'indicated they do not see a path' for Vertex to obtain a broad approval for Journavx in 'peripheral neuropathic pain' — i.e., chronic pain caused by nerve damage or dysfunction. Instead the company will have to obtain approval in individual subsets of chronic pain. Accordingly, Vertex said it will cancel a planned Phase 3 trial in lumbosacral radiculopathy, also known as sciatica, which the company said affects about 4 million people. Journavx had already failed to outperform placebo in a Phase 2 sciatica trial. Instead it will launch a second Phase 3 trial in diabetic peripheral neuropathy, which affects about 2 million people, in the hope of first getting approval in that indication. At the same time, Vertex announced better-than-expected sales for Journavx in acute pain, with more than 110,000 prescriptions filled. Although the pill was hailed as a potential public health breakthrough for providing a non-addictive alternative to opioids, analysts questioned how widely they would be prescribed, given the availability of cheap alternatives. On Monday, Vertex said it earned $12 million from Journavx, compared to consensus Wall Street projections of $6.7 million. Advertisement Vertex also said it has now treated 29 patients with Casgevy, its gene editing therapy for sickle cell disease, including 16 in the last quarter. Casgevy, which has been slow to launch, earned $30.4 million from second-quarter sales.
