Pfizer and Otsuka received bad news in Europe last week when scientific advisors at the European Medicines Agency (EMA) turned down marketing authorisation for their respective drugs.
For Pfizer, it was the second time that the EMA's Committee for Medicinal Products for Human Use (CHMP) declined to recommend its prospective rheumatoid arthritis (RA) treatment Xeljanz (tofacitinib) be approved, following an initial negative opinion in April this year.
Pfizer appealed the decision, but the CHMP confirmed the refusal after re-examination, claiming there were “significant and unresolved concerns about the risk and type of serious infections seen with tofacitinib”.
These risks are related to the drug's function as an immunosuppressant, while the CHMP also noted other side effects, such as liver damage and holes in the gut.
“It was not clear that these risks could be managed successfully in medical practice,” said the Committee, which admitted studies did back the drug's ability to improve the signs and symptoms of rheumatoid arthritis and the physical function of patients.
What wasn't so conclusive was the use of Xeljanz in the consistent reduction in disease activity and structural damage to joints, which the CHMP noted in April.
As part of its appeal, Pfizer proposed to remove claims of an effect on structural damage from the indication, but this was not enough to appease the CHMP due to concerns about the drug's safety.
Dr Steven Romano, senior VP and the head of the Medicines Development Group for Pfizer Specialty Care, said the company was “disappointed” with the CHMP's decision, noting it was made by a “narrow majority”.
“We believe that the benefit-to-risk profile of Xeljanz is favourable, and we remain committed to working with the EMA to make Xeljanz available to appropriate patients in Europe,” he said.
The EMA's decision continues to go against other regulatory bodies, with Xeljanz approved for use in the US, Japan, Russia, Switzerland, Argentina, Kuwait and the United Arab Emirates.
CHMP fails to back Otsuka's delamanid
Pfizer was not the only pharma company to face a disappointing CHMP recommendation, with Japan-based Otsuka receiving a negative option for delamanid in the treatment of multi-drug resistant tuberculosis (TB).
The development of new drugs to combat growing resistance to infections, such as TB, is among the most pressing issues in modern healthcare, but the CHMP turned down delamanid as its benefits had not been sufficiently shown.
Specifically, the CHMP described the two-month duration of treatment as “too short to establish the effectiveness of delamanid”, considering that the drug is intended to be used for at least six months in actual practice.
On top of this, the CHMP also noted that data from follow-up studies was unsuitable for consideration as they only included patients who had agreed to take part, and therefore wasn't representative of typical TB patients.
Compounding this attitude, the CHMP also said that it was unable to determine the most appropriate dosing regimen for delamanid based on the data supplied by Otsuka.
“Otsuka remains committed to the development of its global TB programme and will continue working closely with the CHMP and other regulatory bodies to bring delamanid to market,” said Masuhiro Yoshitake, Otsuka's executive operating officer and TB global project leader.