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Morning Briefing

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Monday, May 3 2021

Full Issue

Newborn Screening Prioritized During Ongoing Pipette Shortage

In other pharmaceutical news, six patients with demential got doses of a gene therapy unapproved in the U.S., by traveling to Mexico; Fox News reports on an "ultra expensive" drug for spinal muscular atrophy; and a lung cancer pill cost is reported by Stat.

Programs that screen newborns for potentially deadly genetic conditions will now have higher priority when ordering pipette tips — a critical laboratory supply that is in shortage. STAT highlighted the pipette tip shortage, which is affecting researchers across the country, on Wednesday. (Sheridan, 4/30)

In other pharmaceutical industry news —

Six patients with dementia traveled to Mexico last year to be injected with a gene therapy not authorized for use in the U.S., according to the CEO of a Seattle-area startup that wants to accelerate testing of unproven anti-aging medicines and views U.S. drug safety regulations as a hindrance. At the heart of the project is a controversial biotech called BioViva, whose CEO had herself injected with an experimental gene therapy in Colombia and whose advisory board includes renowned Harvard geneticist George Church. It is part of a growing ecosystem of entrepreneurs and scientists, dreamers and schemers, who believe aging is not inevitable and aim to develop treatments to extend the human life span. (Molteni, 5/3)

Imagine your child is dying. There's a drug that promises hope, promises to stop your baby's illness dead in its tracks. But this dream medicine is out of reach because it costs around $2 million, depending where you live. This is the story of many of parents of children with Spinal Muscular Atrophy (SMA), a rare motor neuron disease. Between one in 6,000 and 10,000 children born have the disorder in some form. More than two-thirds of those diagnosed with the most common and serious derivation, SMA1, die before the age of 2, if left untreated — and treatment is complex. (Kellogg and Zakrzewski, 5/1)

Investors do not like surprises with negative overtones, so they reacted badly when Amgen announced last Tuesday that it was conducting a new study to compare a 240 mg dose of its KRAS-targeted lung cancer pill, sotorasib, against the 960 mg dose that is now under review by the Food and Drug Administration. Amgen’s stock price fell 6% last week, the most among all the large-cap drug makers. The reaction was mostly interpreted as concern about the drug’s safety. But the real issue and risk for sotorasib could be its price. (Feuerstein, 5/3)

This is part of the Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.
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