In what it called a “victory for public health,” FDA officials Tuesday announced a US District Court decision that upholds the agency’s interpretation of the clinical need for bulk substances that may be used by outsourcing facilities in drug compounding.1

In a statement, Acting Commissioner Norman E. "Ned" Sharpless, MD, and Janet Woodcock MD, director of the Center for Drug Evaluation and Research, said that they are “pleased” with the court’s ruling upholding the FDA's decision to exclude vasopressin from the list of bulk substances that can be used by outsourcing facilities to make drugs, the 503B Bulks List.1 Athenex legally challenged the decision to exclude vasopressin from the 503B Bulks List.2 

“FDA oversight of compounding continues to remain essential, because compounded drugs, while important to patients who need them, pose ongoing and serious risks if they are not manufactured properly. Every year, the FDA learns of cases of patient illnesses and deaths due to improperly compounded drugs and caused by issues like contamination in drugs that need to be sterile because they are entering the bloodstream, the eye, or the spine or because the drugs are not made at the right strength and are super-potent,” the FDA officials said in the joint statement.1

Vasopressin, an antidiuretic hormone, is a nonapeptide that is synthesized in the brain's hypothalamus. It has an impact on the body’s osmotic balance, blood pressure regulation, and proper kidney function.3 

On March 1, 2019, the FDA said that it was excluding vasopressin, as well as nicardipine hydrochloride, from the 503B Bulks List.

In that announcement, then-Commissioner Scott Gottlieb, MD, said that the 2 substances were excluded from the list because the nominator did not provide a medical reason that would explain why FDA-approved drug products containing these substances could not be used or adapted for treatments rather than drugs compounded from bulk substances.

He cited lack of a clinical need for outsourcing facilities to compound drugs from the 2 substances.4

“The law directs the FDA to limit compounding from bulk substances to situations where there’s a clinical need for such products or in situations where the drugs are judged by the FDA to be in shortage. These limits protect patients from unnecessary exposure to the risks associated with drugs compounded from bulk drug substances, while also protecting the integrity of the drug approval process,” Gottlieb said in the March 1 statement.4

Athenex contended that the FDA improperly took into account the availability of a branded drug product when assessing the clinical need of its own product, according to the court.

The company argued that the “clinical need” inquiry be confined to simply asking whether there is a therapeutic use for the bulk drug substance.2

In response to the court's ruling, Jeffrey Yordon, chief operating officer of Athenex, said that the company is disappointed.

“We believe our vasopressin product helps to meet an important clinical need," he said in a statement.5 

Athenex said it plans to appeal the ruling and seek a stay of ruling pending its appeal.

In addition, Athenex will comply with the court's decision by ceasing production and marketing of its compounded vasopressin product until it is permitted to do so.5


References
  1. FDA statement on court ruling related to compounded bulk drug substances and the agency’s ongoing efforts to implement its compounding authorities [news release]. Silver Spring, MD; August 6, 2019: FDA website. www.fda.gov/news-events/press-announcements/fda-statement-court-ruling-related-compounded-bulk-drug-substances-and-agencys-ongoing-efforts. Accessed August 6, 2019. 
  2. United States District Court for the District of Columbia. Memorandum Opinion. Case No. 19-cv-00603 (APM). ecf.dcd.uscourts.gov/cgi-bin/show_public_doc?2019cv0603-37. Accessed August 6, 2019.
  3. Cuzzo B, Lappin SL. Vasopressin (Antidiuretic Hormone, ADH) In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2019-. 2019. 
  4. FDA In Brief: FDA finalizes guidance on evaluating the clinical need for outsourcing facilities to compound drugs with bulk drug substances; provides final decision on two substances [news release]. Silver Spring, MD; March 1, 2019: FDA website. https://www.fda.gov/news-events/fda-brief/fda-brief-fda-finalizes-guidance-evaluating-clinical-need-outsourcing-facilities-compound-drugs-bulk. Accessed August 6, 2019.
  5. Athenex provides an update regarding the vasopressin case [news release]. Buffalo, NY; August 2, 2019: Athenex website. ir.athenex.com/news-releases/news-release-details/athenex-provides-update-regarding-vasopressin-case. Accessed August 6, 2019.