July 31, 2023

The need-to-know this morning

Five elderly people have been blinded by a severe side effect after receiving injections of a newly approved treatment for eye disease from Apellis Pharmaceuticals. The frequency of this side effect — a severe type of eye inflammation — is low but its cause remains unknown. The new safety information related to the Apellis drug, called Syfovre, was presented Saturday by a committee of eye disease experts at the annual meeting of the American Society of Retinal Specialists (ASRS).
Immunogen reported $77.4 million in second-quarter sales of Elahere, its treatment for ovarian cancer. Sales in the first quarter were $29.5 million. The company also announced the departure of Chief Medical Officer Anna Birkenblit.

depression

Sage hopes for a blockbuster with antidepressant

The FDA will decide by Aug. 5 whether zuranolone, a rapid-acting antidepressant made by Sage Therapeutics, passes muster for treating major depressive disorder and postpartum depression. Although some patients experienced a rapid uptick in their symptoms in clinical trials, the studies testing the drug have been a mixed bag. Two trials had positive outcomes, but one was negative, failing to demonstrate a statistically significant improvement in depressive symptoms over placebo.

If approved, however, zuranolone would stand in a class of its own: No approved antidepressants begin to work within three days, like Sage's drug, nor does treatment wrap up after two weeks.

"The idea that you would just take it when you're depressed and stop once you're not, I think, is a paradigm shift, but it's a paradigm shift in an interesting way," a Sage adviser told STAT.

rare disease

Brainstorming gene therapy's tenuous future

Gene therapy has advanced to a point that patients with even "n of 1" diseases could potentially benefit from this technology. However, genetic treatments that have been forged in academic labs and successfully treated a handful of desperate patients often evaporate once it comes time to scale. This science is costly, and doesn't make economic sense for most biotech companies — even if it's technically possible to help or even cure rare and deadly disease. Many promising gene therapies have already been shelved for financial reasons alone.

Gene therapy researchers, advocates, and federal officials met last month in Washington at an invite-only workshop to contemplate this issue. There are about 7,000 recognized rare diseases, and as of the meeting, only seven FDA-approved gene therapies for genetic diseases. "Seven out of 7,000," one researcher said at the event. "We have a lot of work to do." The hope, they say, is to come up with a completely new model for gene therapy development. A "Chipotle" approach has been thrown around — pick your virus, pick your gene, and pick your DNA promoter, and perhaps a scalable gene therapy could be born.

opinion

Why new drug policy might hamper future innovation

Ted W. Love, chair of BIO's board of directors, leads a company called Global Blood Therapeutics that produces a once-a-day tablet for sickle cell disease. He opines for STAT that the current policy shifts are too harsh on the drug industry — and had they been in play previously, his breakthrough sickle cell treatment would have never seen the light of day.

For example, the FTC is cracking down on large acquisitions, particularly in the biopharma sector. Had Global Blood Therapeutics not been acquired by Pfizer, the sickle cell therapy might not have been accessible to patients in lower-income countries.

legislation

Congress divided on approach to drug shortages

The ongoing drug shortages have split Congress: GOP lawmakers have a very different proposed approach than their Democratic counterparts. Draft legislation from Republicans includes language that would make it easier for generic drugmakers to raise their prices to avoid offering steep discounts to hospitals. Republicans are also proposing that drugs prone to shortages be exempt from a Medicaid inflation rebate.

One Democratic congressman described some of these GOP proposals as "handouts" to drug companies, and says they wouldn't help allay shortages. Democrats also think the legislation around shortages should be folded into a larger pandemic preparedness bill that will likely pass — but Republicans want to keep things separate.

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