Every year, hundreds of rare disease patients and caregivers descend on Capitol Hill to participate in Rare Disease Week to learn about federal legislative issues, meet other advocates, and share their unique stories with legislators. As part of this, on March 2, 2017, Hyman, Phelps …
Hardly a day goes by that we don’t see a press release or announcement that a company has requested or received from FDA’s Office of Orphan Products Development (“OOPD”) designation of its drug or biological product as an “orphan drug.” There’s a reason for that: …
By Kurt R. Karst – We recently posted on an orphan drug clinical superiority precedent we came across: PURIXAN (mercaptopurine) Oral Suspension, 20 mg/mL, for the treatment of Acute Lymphoblastic Leukemia in pediatric patients. It’s the sixth “greater safety” orphan drug clincial superiority precedent we know …
By Kurt R. Karst – We like hunting down orphan drug clinical superiority precedents. And although it’s been said that “the thrill is in the chase, never in the capture” (by a Doctor Who character at least), we enjoy the capture just as much as the …
By Kurt R. Karst – Earlier this week, iPR Pharmaceuticals, Inc. (“iPR”), the owner of NDA 021366 for CRESTOR (rosuvastatin calcium) Tablets, 5 mg, 10 mg, 20 mg, and 40 mg, and AstraZeneca Pharmaceuticals LP (“AstraZeneca”), iPR’s agent, filed a Complaint in the U.S. District Court …
By Kurt R. Karst – It was just last week when one orphan drug exclusivity labeling carve-out case (presumptively) came to an end when the U.S. Court of Appeals for the District of Columbia Circuit ruled for FDA in the context of a generic version of …
By Kurt R. Karst – An FDA decision to revoke designation of a drug (or a biological product) as an orphan drug is a rare event. (Though it’s not as rare as revocation of orphan drug exclusivity, which has happened only once – see our previous …
By Kurt R. Karst – On April 27, 2016, Eagle Pharmaceuticals, Inc. (“Eagle”) filed a Complaint in the U.S. District Court for the District of Columbia alleging that FDA violated the Administrative Procedure Act (“APA”) when the Agency refused to grant periods of 7-year orphan drug …
By Kurt R. Karst – For several years now we have been following instances in which FDA designated, and later approved, a drug (including a biological product) as an “orphan drug” on the basis that it is not the “same drug” as a previously approved product …
By Kurt R. Karst – Now that the dust from 2015 has settled, we’re able to take a look at and evaluate the year that was in orphan drug designations and approvals. Our annual review has become quite popular. We often see the numbers we provide …
By Kurt R. Karst – Earlier this week, Public Citizen announced the release of a report, titled “House Orphan Drug Proposal: A Windfall for Pharma, False ‘Cure’ for Patients,” that takes aim at a particular provision in the 21st Century Cures Act (H.R. 6) that the …
By Jennifer M. Thomas & Michelle L. Butler – The U.S. District Court for the District of Columbia ruled decidedly in favor of PhRMA and against the government earlier this week in PhRMA v. HHS, No. 14-1685 (Oct. 14, 2015), potentially concluding a protracted fight between the Health …
By Kurt R. Karst – The legal world is chock-full of tests, doctrines, and processes named after a particular case or court decision. It’s kind of a legal short-hand; a lexicon of legal eponyms. There’s the “Winter Standard” for preliminary injunctions in federal courts; the “Lemon …
By Kurt R. Karst – In a September 10, 2015 Response to a September 2, 2011 Citizen Petition (Docket No. FDA-2011-P-0657) submitted by the National Organization for Rare Disorders (“NORD”), FDA refuses to add a statement to guidance documents concerning orphan drugs that the Agency’s official …
By Alexander J. Varond & James E. Valentine* – FDA recently released a draft guidance, titled “Rare Diseases: Common Issues in Drug Development.” The fourteen-page document amounts to more of a primer than a focused discussion of technical issues. It serves as a useful reminder of the …
