The threat of tariffs on imports of branded drugs is about to be realized after U.S. President Donald Trump announced a 100% import duty will apply beginning Oct. 1. However, the flurry of recent announcements by pharma companies of investments in U.S. manufacturing plants may have paid off, with the president saying in his announcement on Truth Social, that the 100% rate will be enforced “unless a company is building their manufacturing plant in America.” But some may wish they had responded sooner to the possibility of high tariffs, because President Trump defined “is building” as when a project is breaking ground and/or actually under construction. The tariffs leave thousands of small- and mid-sized biotech companies exposed.

Crinetics stock jumps on FDA OK for acromegaly drug

Shares of San Diego-based Crinetics Pharmaceuticals Inc. (NASDAQ:CRNX) were trading at $45.39, up $9.50, or 26%, after the U.S. FDA cleared Palsonify (paltusotine) for the first-line treatment of adults with acromegaly who’ve had an inadequate response to surgery and/or those for whom an operation is not feasible. A selectively targeted somatostatin receptor type 2 nonpeptide agonist, Palsonify is the first once-daily, oral treatment approved for adults with the rare condition caused by excessive growth hormone made by the pituitary gland. Acromegaly leads to abnormal growth in bones, soft tissues, and organs.

Lilly’s SERD in late-stage breast cancer receives FDA approval

Eli Lilly and Co.’s oral selective estrogen receptor degrader (SERD) imlunestrant has been approved by the U.S. FDA for treating late-stage breast cancer patients. Branded Inluriyo, the second-line treatment is for adults with estrogen receptor-positive, human epidermal growth factor receptor-2-negative ESR1-mutated advanced or metastatic breast cancer. The approval was built on results from the phase III Ember-3 study showing the SERD reduced the disease’s progression and death when compared to those receiving endocrine therapy. Inluriyo will be available in the U.S. in the coming weeks, according to Lilly.

MFN pricing model under White House review

As U.S. President Donald Trump promised again in a Sept. 23 media briefing, most-favored-nation (MFN) prescription drug pricing could soon come to America. In keeping with an executive order Trump issued in May, the U.S. Department of Health and Human Services and the CMS began the rulemaking process for an MFN model by filing a proposed rule Sept. 25 to the White House Office of Management and Budget (OMB) for review. The rule would establish the Global Benchmark for Efficient Drug Pricing (GLOBE) Model and is considered to be “economically significant,” according to the OMB website.

Real-world data illuminates Rakuten's Alluminox solid tumor platform

Rakuten Medical Inc. is advancing a pipeline of solid tumor therapeutics built on its Alluminox platform worldwide, having gained conditional early approval (CEA) of ASP-1929, an Alluminox-derived photoimmunotherapy, in Japan in 2020. “It is a new modality,” Minami Maeda, president of San Diego-headquartered Rakuten, told BioWorld on the sidelines of the Asia Bio Partnering Forum in Singapore. “We could say it is like an antibody-drug conjugate (ADC) because it utilizes an antibody … but [it does not have the ADC’s] toxic payload.” Japan’s Pharmaceuticals and Medical Devices Agency cleared ASP-1929 in September 2020 via the country’s Sakigake (fast track) designation and CEA system, allowing patients with advanced head and neck squamous cell carcinoma to receive treatment in clinical trials.

FDA continues to build regenerative medicine pathway

The U.S. FDA released a trio of draft guidances to help sponsors in developing and monitoring cell and gene therapies, as well as other regenerative medicine therapies. When finalized, one of the drafts, “Expedited programs for regenerative medicine therapies for serious conditions,” will replace a 2019 guidance of the same name. The draft described the expedited programs available to sponsors of regenerative medicine therapies for serious conditions, including those products designated as a “regenerative medicine advanced therapy.” It also discussed considerations in the clinical development of those therapies and opportunities for sponsors to interact with CBER review staff.

Also in the news

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