Court ruling in orphan drug lawsuit reframes outlook on market exclusivity
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On Sept. 29, a federal judge dismissed a lawsuit from Catalyst Pharmaceuticals against the FDA and Jacobus Pharmaceuticals over the approval of a rival drug for a rare autoimmune disease — a ruling that disputes the “exclusivity” of the Orphan Drug Act.
In 2018, Catalyst Pharmaceuticals was awarded FDA approval and granted orphan drug status for amifampridine (Firdapse), the first treatment for pediatric patients with Lambert-Eaton Myasthenic Syndrome. Catalyst was under the assumption that market exclusivity was part of the orphan drug designation.
However, in 2019, the FDA also awarded approval and orphan drug status to Ruzurgi, an amifampridine drug from Jacobus Pharmaceuticals.
Catalyst sued. Specifically, the company filed suit against Jacobus in the U.S. District Court for New Jersey and against its partner company PantherRx in the U.S. District Court for the Western District of Pennsylvania. Catalyst’s contention is that Ruzurgi, which is used for pediatric LEMS patients, infringes on the Catalyst patent.
In the ruling, the judge suggested that the Orphan Drug Act may be interpreted in more than one way when it comes to the matter of market exclusivity.
The case raises questions about the 1983 law. The initial intent was to incentivize drug makers to focus attention on treatments for rare diseases and conditions, defined as those that affect fewer than 200,000 people. The statute includes a 50% tax credit for research and development into drugs potentially used to treat such conditions, along with clinical trial subsidies, waived or reduced regulatory fees and eligibility for market exclusivity for 7 years after approval.
While this law has no doubt yielded treatments for conditions that may never have received any R&D attention at all — much less an FDA approval — some experts believe that some pharmaceutical companies have used the act to their advantage by shielding themselves from competition with the promise of market exclusivity. They have turned treatments for rare diseases into multibillion-dollar cash cows.
Catalyst, in fact, came under fire from Sen. Bernie Sanders (D-Vermont) for price gouging, adding a layer of complication to the matter.
Despite the controversies, the success rate of the Orphan Drug Act is indisputable. Since the law took effect, hundreds of companies have developed hundreds of drugs for patients who might otherwise have gone untreated.
Healio Rheumatology sat down with FDA spokesperson Chanapa Tantibanchachai to discuss the history of the Orphan Drug Act and how the 1983 statute works in 2020.
Q. How many drugs of those currently approved under the Orphan Drug Act are for rheumatologic disorders?
Tantibanchachai: Since the passage of the Orphan Drug Act through Sept. 30, there have been 907 approved orphan drug indications. Of these approved indications, 21 have been for rheumatological conditions, which is approximately 2%. Note that certain diseases may be categorized in various ways, and this number represents only one such categorization.
The availability and access of safe and effective medical products for patients with rare diseases is essential to fulfilling FDA’s public health mission. The FDA has implemented the Orphan Drug Act, including the orphan-drug exclusivity provisions, balancing the need to incentivize the development of drugs and biologics for rare diseases with the need to protect innovation and access to these drugs and biologics.
Q. As a specialty with many rare diseases, how has the Orphan Drug Act benefited rheumatology?
Tantibanchachai: The Orphan Drug Act provides financial incentives to facilitate the development of drugs and biologics for rare disease or conditions. These financial incentives include tax credits for qualified clinical trials, waiver of human drug application fees, eligibility to apply for orphan drug grants, and potential eligibility for 7-year orphan-drug exclusivity. These incentives have driven innovation in the rare disease space.
Q. How ironclad are the incentives provided by orphan drug status?
Tantibanchachai: The FDA has implemented the Orphan Drug Act through carefully considered regulations that aim to provide the benefits of orphan-drug designation while limiting potential misuse from industry.
Q. Does orphan drug status exclude other, potentially better, drugs coming before the FDA?
Tantibanchachai: It is important to distinguish between orphan-drug designation — also known as “orphan status” — and orphan-drug exclusivity. Orphan-drug designation alone does not prevent the FDA from granting orphan-drug designation or marketing approval to any other sponsor in any way, whether or not the drug is the same or different, and whether or not the intended disease is the same or different.
Under orphan-drug exclusivity, the FDA may not approve another sponsor’s application for the same drug for the same indication, unless the subsequent drug is clinically superior to the drug covered by exclusivity. Therefore, orphan-drug exclusivity does not block the approval of a different drug for the same indication and does not block the approval of the same drug for a different indication. And orphan-drug exclusivity will never block approval of a clinically superior drug that offers a significant therapeutic advantage over and above that provided by the already approved drug.
Q. What proportion of new medicines on the market are orphan drugs?
Tantibanchachai: From Jan. 1, 2015, to Sept. 30, the FDA has approved 265 novel drugs and biologics, and 128 have been approved for at least one orphan indication (48%).
For more information:
Chanapa Tantibanchachai can be reached at 10903 New Hampshire Ave., Building 32, Room 5245, Silver Spring, MD 20993; email: Chanapa.Tantibanchachai@fda.hhs.gov.
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