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 …
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Orphan Drugs
- Orphan Drug Designation Requests and Designations Dipped in 2018, But Orphan Approvals Are Up Again!March 28th, 2019
- 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 …
- Fitting New Scientific Advances Into an Old Regulatory Paradigm (Part 2): Gene Therapy and Orphan Drug “Sameness”July 26th, 2017
Earlier this week, we discussed in a post how FDA, under the Agency’s decades-old regulations defining the term “same drug,” evaluates orphan drug “sameness” in the context of fusion proteins. At the end of that post we suggested that gene therapy “sameness” might be the …
- Fitting New Scientific Advances Into an Old Regulatory Paradigm: Fusion Proteins and Orphan Drug “Sameness”July 25th, 2017
FDA’s Orphan Drug Program, which traces its birth back to the January 4, 1983 enactment of the Orphan Drug Act (“ODA”) (see our previous post here), is probably one of the most successful FDA programs to date. The success of the ODA is most apparent …
- FDA Determines that Deuterated Compounds are NCEs and Different Orphan Drugs Versus Non-deuterated VersionsJuly 16th, 2017
Back in mid-May 2017, when FDA issued the fourth cumulative supplement to the 37th edition of the Orange Book, observant readers may have noticed a couple of interesting entries on page A-7 of the publication. There, in the “PATENT AND EXCLUSIVITY DRUG PRODUCT LIST,” FDA …
- FDA User Fee Package Includes “Technical Corrections” to Address Orphan Drug Clinical SuperiorityMay 9th, 2017
Earlier this week, the U.S. Senate Health, Education, Labor, and Pensions (“HELP”) Committee released a “Manager’s Amendment” to S. 934, the FDA Reauthorization Act of 2017 (“FDARA”). The 171-page Manager’s Amendment, which is slated for a HELP Committee vote later this week, includes several riders …
- Orphan Drugs: The Current Firestorm, a Real Evergreening Issue, and a Possible SolutionMarch 12th, 2017
Periodically, legislators and others become concerned about reports citing the high price of some orphan drugs, including drugs that achieve blockbuster status (earning more than $1 billion a year). Several proposals have been introduced in response to such concerns. In 1990, Congress passed legislation that …
- Rare Disease Week Recap: HP&M’s Frank Sasinowski Briefs the Rare Disease Congressional CaucusMarch 6th, 2017
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 …
- Orphan Drug Approvals and Designations Dipped in 2016, But Orphan Drug Designation Requests SkyrocketedJanuary 25th, 2017
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: …
- The Third Time’s the Charm for RAVICTI and Orphan Drug Designation: Another “Greater Safety” Clinical Superiority PrecedentJuly 25th, 2016
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 …