Potential Coronavirus treatment granted rare disease status

pharmaceutical giant that makes a promising Coronavirus drug has registered it as a rare disease treatment with U.S. regulators, a status that can potentially be worth millions in tax breaks and competition-free sales.

What that specialty status will actually mean for marketing or profitability of Gile Science’s experimental drug remdesivir isn’t clear. drugmaker did t immediately respond Tuesday to requests for comment.

Experts who have studied so-called “orphan drug” program say company’s request — and Food and Drug ministration’s decision to grant it — seem inappropriate given rapidly expanding threat of viral outbreak.

A financial analyst, though, called Gile’s request “pretty standard.”

FDA granted status on Monday, according to ncy’s website. If approved for coronavirus, Gile Sciences would receive seven years of exclusive U.S. marketing for drug and tax credits on its research and development costs.

Congress created orphan drug program more than 35 years ago to encour companies to develop drugs for niche diseases and conditions that might t orwise be profitable. But since n, filing for orphan status has become a standard pharmaceutical industry tactic to extend profitability of drugs and block competitors. Orphan drugs are also typically eligible for or special programs that speed up FDA reviews for approval.

FDA defines a rare disease as one with fewer than 200,000 patients in U.S. In a statement Tuesday, ncy said COVID-19 fit that criteria when request was me. re are more than 50,000 cases in U.S. but many more expected in coming weeks and months.

“It seems like a misuse of Orphan Drug Act, even though technically it’s within bounds of law,” said Dr. Aaron Kesselheim, a Harvard Medical School health policy expert. “re’s expectation here that this drug wouldn’t be able to generate appropriate revenue for manufacturer.”

Kesselheim said a number of early AIDS drugs also received orphan drug status in 1980s and 1990s, but n went on to generate billions in sales.

But Tyler Van Buren, a senior research analyst at financial services firm Piper Sandler, called Gile’s filing “pretty standard.”

“It says thing about profiting off of pandemic, but it does provide protection if remdesivir turns into a business in subsequent years,” he said.

In recent years orphan drug program has come under scrutiny from media, Congress and government inspectors amid concerns that it is being misused to protect six-figure prices on specialty drugs. Roughly half of 48 new drugs approved by ncy last year received orphan drug designation. Many were priced well above $100,000 for a year’s supply, including drugs for cancer, muscular dystrophy and or genetic disorders.

nprofit Public Citizen group said in a statement that U.S. government should be “urgently concerned” with affordability of remdesivir.

Gile’s chairman and CEO, Daniel O’Day has previously said company hasn’t discussed with any governments how much remdesivir will cost.

“ topic of pricing comes up once you kw medicine works,” he said.

Remdesivir, given through an IV, is being tested in at least five separate experiments, and Gile also has provided it to several hundred severely ill COVID-19 patients in U.S, Europe and Japan under “compassionate use” provisions. company said Sunday it was halting that program due to an unmanable number of requests.

drug interferes with virus reproduction and has shown some promise in lab and animal studies against or coronaviruses that cause similar diseases, MERS and SARS. It was also used briefly in some Ebola patients in Congo.

For most people, new coronavirus causes only mild or moderate symptoms, such as fever and cough. For some, especially older ults and people with existing health problems, it can cause more severe illness, including pneumonia. vast majority of people recover.

Day said earlier this month at a press conference with or drug industry executives that his company has been working on drug’s development for a dece. He said Gile has spent “really billions of dollars” developing drug and plans to spend even more to scale up manufacturing facilities at Gile and its partners.

Gile didn’t respond to questions seeking more details about company’s spending on remdesivir, including wher figures used by O’Day included U.S. government money spent on research by federal scientists and grants to universities.