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.

Stat:
HHS To Prioritize Newborn Screening Programs’ Pipette Tip Orders 

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 —

Stat:
Six Dementia Patients Got An Unapproved Gene Therapy, CEO Says 

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)

Fox News:
Ultra-Expensive Medicine A Desperate Need For Thousands Of Babies Around The World

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)

Stat:
With Amgen’s KRAS Cancer Drug, Worry About Pricing, Not Safety

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)

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