Our last post on the Eleventh Circuit’s September 2021 decision in Catalyst v. Becerra got a lot of attention. We’d like to think that this is because the scope of orphan drug exclusivity is as fascinating to everyone as it is to us, but if …
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Orphan Drugs
- Higher and Higher (Into the Fire): Jacobus Appeals Orphan Drug Case to SCOTUSApril 21st, 2022
- Condition Critical: Court Interprets Orphan Drug Exclusivity BroadlyFebruary 17th, 2022
Because a drug is designated an “Orphan” if it is intended to treat a “rare condition,” the condition itself always has been integral to Orphan Drug Exclusivity. Indeed, the condition for which the product is intended to treat dictates the prevalence calculation by which FDA …
- Oops!… [FDA] Did It Again: Another Orphan Drug Act Loss for FDA Based on Unambiguous Statutory Text; 11th Circuit Rules that the Scope of Orphan Drug Exclusivity is Determined by the Rare Disease or Condition Designated, and Not the Indication ApprovedOctober 5th, 2021
In the appellate courts lately, it’s been FDA “Against the Music.” In yet another decision based on statutory interpretation, an appellate court has decided that FDA’s interpretation of the Federal Food, Drug, and Cosmetic Act (FDCA) is contrary to the plain text of the statute. …
- HP&M’s Frank Sasinowski Receives “2021 Pontifical Hero Award for Inspiration” from Pope Francis & Vatican for his Many Decades of Rare Disease AdvocacyMay 12th, 2021
Hyman, Phelps & McNamara, P.C., is proud to announce that Frank J. Sasinowski, a Director at the firm, this past Saturday was awarded by Pope Francis the “2021 Pontifical Hero Award for Inspiration” for his “unwavering advocacy for those who live with rare diseases.” The award …
- Industry – 3, FDA – 0: Will the Agency Finally Throw in the Towel?April 3rd, 2020
In a March 13, 2020 opinion, the United States Court of Appeals for the District of Columbia Circuit handed Eagle Pharmaceuticals, Inc. (Eagle) a significant win in FDA’s appeal from a District Court order requiring FDA to grant Orphan Drug Exclusivity to Eagle’s Bendeka (bendamustine) …
- HP&M Attorney Co-Authors Nature Reviews Article with NIH, FDA, and EMA on the Progress and Challenges of Drug Development for Rare DiseasesDecember 19th, 2019
On December 13, 2019, Nature Reviews Drug Discovery published an article that reviews the landscape of the development of drugs for rare diseases, both the progress and challenges. The article was authored by HP&M Attorney James E. Valentine along with co-authors from the BioPontis Alliance …
- Everything Old is New Again: FDA Revises its 2014 Rare Pediatric Disease Priority Review Voucher Guidance for IndustryAugust 2nd, 2019
Developing drugs for kids with rare diseases is important work. If you would like to hear from one of these kids, check out this video: “My Philosophy for a Happy Life,” by the late Sam Berns. Offering priority review vouchers to sponsors that develop new drugs …
- HP&M Shares Experience with 16 Externally-Led Patient-Focused Drug Development Meetings; Summarizes Commonalities in Rare Disease Patient Perspectives in Comment to FDAMay 31st, 2019
On April 29, 2019, the Food and Drug Administration (FDA) convened a public meeting entitled “Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities.” This meeting was attended by people with rare diseases, their caregivers and family members, patient advocates, industry representatives and …
- Orphan Drug Designation Requests and Designations Dipped in 2018, But Orphan Approvals Are Up Again!March 28th, 2019
Okay, okay . . . . It’s now a month after Rare Disease Day (on which Hyman, Phelps & McNamara, P.C.’s own Frank J. Sasinowski took part in some of the festivities). We’re a little late this year in pulling together and posting on the …
- Orphan Report: The GAO’s Report on Orphan Drug Designations and ApprovalsDecember 6th, 2018
While riding out the end of this term, the Government Accountability Office (GAO) delivered to our lame duck Congress some light reading on orphan drug designations and marketing. In a report titled “FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue,” the …
- Celebrating the Orphan Drug Act’s 35th Anniversary: HP&M Attorneys Author Proposal for Building an FDA Rare Disease Center of Excellence in Advance of EveryLife Foundation Scientific WorkshopSeptember 12th, 2018
As we celebrate the 35th Anniversary of the Orphan Drug Act (see our 30th anniversary post here), periodic consideration of opportunities to reform and refine the approach to rare disease medical product regulation is warranted – similar to the review that occurred 10 years ago, which …
- Going Strong! Orphan Drug Approvals and Designations Skyrocketed in 2017, While Orphan Drug Designation Requests Dipped SlightlyFebruary 28th, 2018
February 28, 2018 is Rare Disease Day. In honor of that day, we thought it would be the perfect time to provide our annual rundown of the past calendar year in orphan drug designations and approvals. And what a year 2017 was for orphan drugs! …
- FDA Implements FDARA Provision on Orphan Drug Clinical Superiority DeterminationsFebruary 26th, 2018
Obtaining FDA decisions detailing when one orphan drug has been determined by the Agency to be “clinically superior” to another orphan drug – either to obtain approval of a product notwithstanding another company’s orphan drug exclusivity for the same drug for the same indication, and/or …
- FDARA Enacted; HP&M Issues Detailed Summary and AnalysisSeptember 7th, 2017
On August 18, 2017, President Trump signed into law the Food and Drug Administration Reauthorization Act of 2017 (“FDARA”). In addition to reauthorizing and amending several drug and medical device provisions that were scheduled to sunset, FDARA also makes several changes to the law concerning …
- United Therapeutics Sues FDA After Agency Denies Orphan Drug Exclusivity for ORENITRAMAugust 9th, 2017
In a Complaint filed in the U.S. District Court for the District of Columbia last week, United Therapeutics Corporation (“UTC”) alleges that FDA unlawfully denied granting the company a period of orphan drug exclusivity upon the Agency’s December 20, 2013 approval of NDA 203496 for …