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The new approval brings acromegaly patients the choice of a once-daily pill that also gives its developer, Crinetics Pharmaceuticals, the opportunity to compete with and take market share from blockbuster products that are well-established in the acromegaly space.

The drug, paltusotine, is the first commercial product for Crinetics, which specializes in developing therapies for endocrine disorders.

The regulatory decision covers the pill’s use as a first-line acromegaly treatment, putting it on par with the first-line injectable peptides currently sold by Novartis and Ipsen. (MedCity News)

The FDA approved the oral estrogen receptor antagonist Inluriyo (imlunestrant) for previously treated ESR1-mutated advanced/metastatic breast cancer.

The approval stipulates use in adults with estrogen receptor-positive/HER2-negative breast cancer that has progressed on at least one line of endocrine therapy.

Inluriyo joins Orserdu (elacestrant) as the only FDA-approved oral therapies for ESR1-mutant breast cancer.

In contrast to Orserdu, which is approved for use in postmenopausal women, imlunestrant’s approval is not limited by menopausal status. (MedPage Today)

The FDA expanded the approval of Regeneron Pharmaceuticals’ fully human monoclonal antibody, Evkeeza (evinacumab-dgnb), for the treatment of HoFH in children aged between one and four.

The approval allows Evkeeza to be used as an adjunct with dietary adjustments, exercise and other lipid-lowering therapies.

The decision to broaden the antibody’s indication was based on clinical efficacy and safety data from six children with HoFH who participated in either the US expanded access program or the compassionate use program outside the US.

HoFH is the most extreme form of familial hypercholesterolemia. (Pharmaceutical Technology)

One upcoming verdict, for instance, could mark another industry first for CAR T therapies, while another could mean the comeback of a previously-pulled cancer drug.

Commissioner Marty Makary tried to calm employees’ fears about firings in a video earlier this week, saying he was able to “ensure an exemption because we are in the business of public safety.”

That could affect Moderna, Avidity Biosciences, Viridian Therapeutics, Cogent Biosciences, and Vera Therapeutics, which were all expected to complete their filings before the end of this year. (STAT)

The site, intended to launch next year, functions as a sidecar to recent White House agreements with drugmakers meant to cut US costs.

The Wall Street Journal reported that most insured patients probably pay less through their plans than they would at cash prices on TrumpRx.

Pfizer plans to offer nearly all drugs to Medicaid at “most-favored-nation” prices, and Pfizer agreed it won’t charge more in the US for new drugs compared to other wealthy countries.

Pfizer said its drugs will be available through TrumpRx at discounted rates, but only for patients not using insurance.

Pfizer said it will also invest $70 billion in US manufacturing and research. The drugmaker will also be exempt from tariffs — which Trump has threatened on companies that don’t come to the table on drug prices — for three years.

And Medicaid already pays some of the lowest prices for drugs, and most on Medicaid pay little to no money out of pocket.

The site will not sell or distribute medications directly.

Instead, customers can search for medications on the site, CNN reports.

They will then be redirected to a direct-to-consumer channel from the manufacturer to make purchases, officials said. (Axios)