Back in January, Takeda won approval for a new indication for its primary immunodeficiency drug HyQvia, this time as a treatment for a rare autoimmune disorder called chronic inflammatory demyelinating polyneuropathy.
Now, the FDA is warning a doctor who worked on part of the clinical package that helped win the approval. A warning letter published Tuesday, which was sent in May to one of the doctors who worked on the Phase 3 trial, highlights several deficiencies found in a 2023 inspection dealing with the way he conducted his portion of the trial.
The FDA took issue with Konrad Rejdak, the Poland-based doctor, and his comments that part of the problem with the trial was poor internet connection during the pandemic. As a result, some of the assessment data for the placebo-controlled trial were entered into tablets “at a later time, either based on written notes or on their memory.”
The warning letter notes that the doctor’s response doesn’t concur with what was relayed during the inspection, and that all affected trial visits occurred before the pandemic.
Rejdak also told the FDA that trial data could be entered into tablets in “offline mode” and uploaded once an internet connection was established.
“We find this explanation inadequate because this modality could not be demonstrated during the inspection,” the FDA said.
Takeda acquired HyQvia as part of its $62 billion takeover of rare disease specialist Shire in 2019. The company’s Phase 3 trial ended in 2022, and Takeda said it met the primary endpoint in June of that year. The trial of 138 CIDP patients was run in 23 different countries.
Takeda said the approval package is not in question, adding in a statement, “We collaborated with the FDA to address the findings, and our approved prescribing labels for HYQVIA and GAMMAGARD LIQUID ultimately excluded efficacy data from this clinical trial site. We are confident in the safety and efficacy analyses from our ADVANCE-CIDP clinical research program, which are based on results from more than 80 different research sites around the globe.”
Editor’s note: Article updated with Takeda comment.