Fabry patients petition NIH on shortage

Frustrated Fabry disease patients are again trying to persuade U.S. officials to open up competition for Genzyme's patented Fabrazyme therapy. The only drug approved in the U.S. to treat their rare disorder, Fabrazyme has been in short supply for more than a year since viral contamination forced a shutdown at Genzyme's key plant.

The patient group's attorney filed a petition with the National Institutes of Health, asking for a rehearing of their plea to allow other drugmakers a crack at making the patented drug, the Pittsburgh Post-Gazette reports. The NIH denied that request in December, maintaining another manufacturer wouldn't be able to alleviate the short-term scarcity of the drug; it would simply take too long to gear up for manufacturing the complex drug.

But the patients' new petition says Genzyme's drug is still scarce--and the company has pushed back its target date for getting back to full strength. That isn't expected until year's end, the petition says. In the meantime, Fabry patients have had to accept lower doses and skip some infusions altogether.

Some of the petitioning Fabry patients have also sued the company for increased access to the drug. Genzyme asked the judge in that case to dismiss the claims or move them to Massachusetts federal court. "We know this is a very difficult time for the patient community," spokeswoman Lori Gorski told the newspaper, "and we share a common goal of returning an uninterrupted supply Fabrazyme as quickly as possible."

- read the Post-Gazette piece

Suggested Articles

An oncologist leader RBC Capital Markets interviewed expressed comfort that Trodelvy will succeed in earlier-line TNBC and in HR-positive disease.

Gilead Sciences is the latest drugmaker to settle Justice Department claims that its charity contributions were actually kickbacks.

J&J figures its partner Genmab owes a share of Darzalex Faspro royalties to Halozyme for its subcutaneous delivery tech. Genmab doesn't agree.