In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups offered solutions to ease the impacts if the plan must go ahead.
Despite billions being committed to reshoring pharma manufacturing in the face of tariff threats from President Donald Trump, industry does not seem prepared to entirely forgo foreign operations. In particular, rare disease biotechs and those developing complex modalities like cell and gene therapies have urged the Trump administration to provide exemptions to pending pharma tariffs.
The pleas come in the form of comments filed over the past few weeks on the government’s Section 232 investigation being conducted to determine if tariffs can be levied against pharma companies as a matter of national security.
“With 95% of the more than 10,000 known rare diseases still lacking an FDA-approved therapy, it is in the interest of the public health to preserve incentives that sustain this innovation ecosystem for rare diseases,” BioMarin wrote in its seven-page letter. The company has a handful of approved gene therapies such as Roctavian for hemophilia A as well as new medicines for diseases that have long lacked options, but has struggled to break even.
BioMarin’s is one of hundreds of comments posted last week via the Federal Register. Combing through them reveals the broad issues that companies and industry groups have with the proposed import taxes.
The Alliance for Regenerative Medicine (ARM) said that tariffs could undermine and slow access to cell and gene therapies for U.S. patients. If tariffs are put into place, ARM urged the administration to do so in a phased approach that would provide time for companies to reshore production. Small volume imports could also be exempted, the group said.
ARM explained that regenerative medicines are sometimes produced for just a handful of patients in a year and demand can be highly variable. But when they are needed, the situation is often time-critical, meaning companies require certainty that they can access the needed ingredients, which often come from outside the U.S.
“To ensure patient access, manufacturers must have confidence that they can secure ingredients from the widest possible set of reliable sources, at a predictable cost,” the group said.
ARM asserted that even though the patient population for cell and gene therapies is small, losing access to them would be devastating. “Disrupting access to these therapies will cut off some of the most vulnerable and complex patients in our healthcare system from life-changing treatment,” the group’s comment read.
The Rare Disease Company Coalition similarly advocated for its patient community by requesting that orphan drugs and their components be exempted from tariffs. The group is run by executives from rare disease companies including Fulcrum Therapeutics, AstraZeneca’s rare disease subsidiary Alexion, argenx, Travere Therapeutics, Sarepta Therapeutics and Inozyme Pharma.
“Efforts to rapidly reshore the entire supply chain for orphan drugs, including those in development and those already approved, would be economically prohibitive,” RDCC wrote. “Orphan drug manufacturers lack economies of scale and often depend on highly specialized overseas partners for producing and packaging the final dosage form.”
BioMarin said it has recently become profitable after years of reinvesting the bulk of its revenue back into R&D. This was possible thanks to the Orphan Drug Act, which was enacted by Congress with the goal of ensuring that all trade policies support incentives for orphan drug development. The company requested that orphan drugs be excluded from potential tariffs.
“Tariffs on these finished products and critical inputs would have an immediate and disproportionate effect on the availability and affordability of orphan drugs,” BioMarin wrote. “Increased costs and supply disruptions could delay treatment development, constrain manufacturing capacity, and threaten timely access to life-saving medicines for patients who have no therapeutic alternatives.”
Complex Cancer Drugs
Perspective Therapeutics, a biotech developing radiopharmaceuticals, also asked for leniency considering the highly specialized manufacturing process for its complex radiation therapeutics.
While some of the comment from CEO Thjis Spoor was blacked out, he asked the Trump administration to defer tariffs on the specialized equipment needed to manufacture its radiopharmaceutical cancer therapies. The company already conducts most of its work in the U.S., having spun out of research at the University of Iowa and operating two stateside manufacturing sites.
Japanese biopharma Daiichi Sankyo touted the success of its AstraZeneca-partnered cancer antibody drug conjugate (ADC) Enhertu, and follow-up drug Datroway, as success stories that have benefitted cancer patients in the U.S. But as with radiopharmaceuticals, ADCs are complex to manufacture, with the potential for batch-to-batch variations.
Enhertu is manufactured at facilities in the U.S., Japan and Europe, then packaged in Ohio before warehousing and distribution from Tennessee.
“Moving the overseas manufacturing steps to the U.S. would require significant overhaul of the company’s operations and substantial financial costs,” Daiichi explained.
The company is already moving some additional processes to the U.S. in the coming years, but the second step—production of the conjugated product—is done overseas and would be hit by tariffs if imposed.
“The application of tariffs to Enhertu and Daiichi Sankyo’s other medicines risks real consequences on the company’s ability to meet its goals for significant investment in the future of new treatments for patients and planned manufacturing capacity in the U.S.,” Daiichi wrote.
