We just posted yesterday: “Redesivir was approved by the FDA as an orphan drug “ article, a major reversal occurred today:” On March 26, Gilead Sciences submitted an application request to the US FDA , Revokes its orphan drug status granted to remdesivir for the treatment of COVID-19, and renounces all preferential rights related to this qualification. “This article will explain why Gilead suddenly did so Big change? What will happen in the future?
This article is from the WeChat public account: Chang Gung Medical Research (ID: cghmstudy) , author: HA is the division, from the head of FIG: IC photo
Gilead (Gilead) The company’s Red Desivir (remdesivir ) won the FDA’s (COVID-19) Indication of orphan drug for indications (It is only identified as an orphan drug and the drug has not been approved) triggered There is widespread concern.
Today, Gilead issued a statement saying that it has submitted an application to the FDA requesting the FDA to revoke the orphan drug designation of Redecive and waive all rights and interests related to the orphan drug designation. Kyrgyzstan Lead expressed confidence in expediting the regulatory review of Redecide without orphan drug designation.
Gilead also specifically stated in the statement that one of the benefits of orphan drug identification is that it can waive the requirement to provide a pediatric research plan before the new drug application is filed. The review process may take up to 210 days. .
The orphan drug identification puts Redeevir into controversy
Since Redecive has been identified as an orphan drug, there have been various objections to public development of various organizations, especially patient rights advocacy organizations, all hoping that the FDA revoke the relevant identification. This controversy focuses on a few points:
Whether the first government engages in the delivery of benefits through unseen contacts with pharmaceutical companies, such as Patients Over Pharma called for the immediate revocation of this determination. The organization’s focus is to expose the Trump administration and the pharmaceutical industry. contact.
Second, it is considered that such a finding lacks sufficient evidence. For example, the director of Knowledge Ecology International (Knowledge Ecology International (KEI) said, His team may try to convince the FDA that there is insufficient evidence to identify the orphan drug. He stated that KEI may first file a citizen petition requesting the determination to be revoked.
Third, worrying about the harm to public interests caused by the privatization of drugs. This view is represented by Democratic presidential candidate Bernie Sanders. (Bernie Sanders) , he believes that “after the taxpayer invested tens of millions of dollars to develop Radixivir, Trump’s FDA is using the laws set for rare diseases to privatize drugs for pandemic diseases , This is really outrageous.
65 years ago, when Jonas Salk developed the polio vaccine, he knew it would be of great value to all humans, so he refused to apply for a patent. Now we must put human life above corporate profits. We cannot allow pharmaceutical companies to monopolize drugs that can save millions of people in this crisis. “
Can a dispute be eliminated by reversing the determination? Really not. Although the stimuli brought to society by orphan drugs are positive and negative, if you revoke the identification, you won’t be applauded! Public citizens (Public Citizen) The organization is keeping up with it and publicly claims that Gilead:
“Today’s so-called withdrawal is not enough. If Redecive is proven to be a viable drug for COVID-19, then the world cannot allow a pharmaceutical manufacturer to monopolize the drug, especially considering In the case of a large amount of public investment in this drug, Gilead is required to do more than just make vague and reasonable pricing commitments. It should immediately commit to licensing all the pharmaceutical manufacturers’ rights to Redecive and the required Technology in exchange for a modest concession. “
Is orphan drug identification basis, can it be revoked?
FDA’s orphan drug designation is designed to help companies develop and provide incentives for minority populations (up to 200,000 in the United States) Measures such as tax credits for research and a seven-year orphan drug franchise for drug sponsors. Although the number of COVID-19 cases in the United States is currently less than 200,000, some people are beginning to criticize this finding as an abuse of the orphan drug designation.
21 CFR 316.29 Regulatory provisions regarding the withdrawal of orphan drug determinations: The FDA may revoke orphan drug determinations for any drug if the FDA finds the following:
(1) The assertion request contains untruthful material factual statements; (2) the assertion request omits substantive information required by regulations; (3) FDA subsequently discovered that when the request was made, the drug was not actually Eligible for orphan drug certification.
According to these revocation requirements, Kat is not yet knownWhat is the specific reason for Lead’s withdrawal of the application, but in any case, the easiest to determine is that the orphan drug has been abused and the eligibility problem has occurred. In fact, this is not the first time that Radixivir has been certified as an orphan drug. Ridoxivir was certified as an orphan drug in 2015 for the treatment of Ebola indications.
Aphidicolin, pyrrolidine tetraphosphate, quinacrine, and ranpimase have also received orphan drug designation for Ebola. According to the regulations revoking the orphan drug designation, even if the number of COVID-19 patients exceeds the 200,000 threshold (as of the date of publication, there are about 55,000 confirmed patients in the United States) will not revoke the orphan drug designation of Redecive. Because according to a Gilead spokesperson:
“On request for orphan drug identification, only a few Americans were affected by COVID-19.”
Effects of withdrawal: pricing and franchise period
The price of orphan drugs can be set very high. The average market price of orphan drugs in 2017 was $ 186,758. Bernstein biotechnology analyst Ronny Gal said in an investor report on Monday that if the drug is approved and priced as high as other similar treatments, (about $ 5,000) , and if used only for inpatient intensive care, Gilead could end up with $ 11 billion in revenue.
Gilead may over-price Red Desieve, but there is also a lot of pressure to not do so. Given the number of people who may be infected in the United States, Gilead may not need to do this. But more importantly, the orphan drug determination may deny the U.S. government to increase the supply of antiviral drugs (if it proves effective) All efforts for patent rights related to Redecive.
Gilead replied:
“In response to this global health emergency, Gilead has been investing significantly in venture capital and researching Red Seaway as a potential COVID-19 The safety and effectiveness of therapeutics in order to provide the urgent use of Redecive in a responsible manner while conducting these studies and to maximize the scope of their supply. If Redecive is proven to be a safe and effective treatment for COVID-19, we will work to make it available and affordable to governments and patients around the world. “
Because all the patent terms of the therapeutic drug exceed the seven-year franchise period of the orphan drug designation (This means that over the seven-year period There will be no generics on the market) , so if the drug is approved, another benefit of Gilead’s orphan drug designation is its 25% tax credit for research and development.
There are currently five clinical trials of Radixavir, Gilead is responsible for two, and the company also said that it is producing two Radixavir preparations (Liquids and lyophilizates) , is expanding its network of production partners and has begun in-house production to supplement Redecive’s capacity.
As concerned, it is unknown how Gilead will price if Rhedivevir is approved as an orphan drug? Even if Gilead’s application for the withdrawal of orthopedic drug Redecide is approved, if it is effective against the new crown virus, what will the true price of Redecive be? This is what people are worrying about now!
This article is from the WeChat public account: Chang Gung Medical Research (ID: cghmstudy) , author: Medical tubes division.