FDA Promises Flexibility for Stem Cell Trials

February 18, 2019

Regulators are promising “flexibility” in clinical trial design for stem cell therapies in rare diseases.

In a final guidance issued last week, the FDA says it “recognizes that, for regenerative medicine therapies for rare diseases, certain aspects of drug development that are feasible for common diseases may not be feasible, and that development challenges can be greater with increasing rarity of the disease.”

“Innovative trial designs, such as trials that compare several different investigational agents to each other and a common control, may be particularly useful in studies of regenerative medicine therapies to treat rare diseases,” the guidance says.

CBER might also be open to historical controls data obtained from natural histories, and regulators might be willing to consider a trial that — unlike the traditional, multi-site study — uses multiple sites that are sharing data on stem cell therapies in a combination to support a BLA.

Sponsors are encouraged “to obtain input from the affected patient communities” when they’re coming up with endpoints for stem cell trials.

The FDA also is promising to be flexible about fast-tracking some potential stem cell treatments. The agency will want clinical data to justify a fast-track application, but the data doesn’t necessarily have to come from prospective clinical trials.

“In some cases, clinical evidence obtained from clinical investigations with appropriately chosen historical controls may provide sufficient preliminary clinical evidence of the potential to address an unmet medical need,” the agency says. “In other cases, preliminary clinical evidence could come from well-designed retrospective studies or clinical case series that provide data systematically collected by treating physicians.”

Read the guidance here:

FDA Issues Draft Guidance on Evaluation of Devices Used With RMATs
New guidance issued by the FDA on Friday outlines the conditions under which clinical evidence will be required in a marketing authorization application for a device used in recovery, isolation or delivery of regenerative medicine advanced therapies (RMATs).

In cases in which sponsors submit separate applications for the RMAT and the device, they have the option to streamline or simplify the regulatory process. “For example, when appropriate and legally permissible, clinical or performance data generated in association with studies of one product may be submitted to support certain aspects of the approval or clearance of other, related product applications,” the guidance states. In other cases, it may be possible for one regulatory submission to refer back to existing performance data from another submission when available.

In cases where a device may be intended for use with only one particular type of cell-based RMAT or with more than one type, it may be necessary to repeat the testing process to assess interactions between the two components for each combination. Sponsors should provide detailed and specific justifications for the applicability of the data and provide sufficient compatibility data ahead of clinical trials.

Read the draft guidance here:

FDA Gives Priority Review to Keytruda
Merck has won priority review for its blockbuster anti-cancer drug Keytruda after promising data from a pivotal Phase III trial, the company announced last week.

The FDA granted priority review for Merck’s supplemental BLA for its blockbuster immunotherapy Keytruda (pembrolizumab) for head and neck cancers. It’s already approved as a second-line treatment for head and neck cancer and several other forms of cancer. The drug has shown “significant improvement” for head-or-neck cancer patients’ overall survival in a Phase III trial, the company said.

The FDA committed to completing the priority review no later than June 10.

Novartis’ Fevipiprant Makes Asthmatic Muscles Smooth, Researchers Find
Novartis’ anti-asthma medication Fevipiprant appears to be the first anti-asthma drug ever to smooth out the muscles lining patients’ airways and may be the first new asthma drug to be approved in 20 years.

Fevipiprant is already in a Phase III trial involving nearly 1,600 patients, scheduled to wrap up by 2022. Researchers from England and Canada, though, crunched the data behind the drug’s last trial, a Phase IIa trial that involved 61 patients from 2012-13.

They found that, like most asthma medicines, Fevipiprant helped ease inflammation in the lungs of its patients, but it also reduced the amount of muscle in the lining of the lungs. An increase in smooth muscle is the strongest predictor of restricted airflow.

Texas Women’s Clinic Signs Up for Trials Network
The Corpus Christi Women’s Clinic has agreed to join Elligo Health Research’s clinical trials network, the clinic announced last week.

Elligo, a patient recruitment company, offers Corpus Christi Women’s Clinic “the infrastructure… necessary to conduct research in its office, effectively keeping patients closer to home and under the care of their trusted physician,” the clinic said in a news release.

Under the agreement, several of the clinic’s doctors have agreed to offer themselves as principle investigators in clinical trials.

Charles River Spends $510 Million for Citoxlab
Charles River Laboratories has agreed to acquire private safety watchdog Citoxlab for nearly $510 million, the companies announced last week.

The acquisition gives Charles River — which claims to have participated in 85 percent of successful drug trials last year — a larger footprint in Europe. Citoxlab offers an array of early-stage services, from specialized toxicology to genomics.

Charles River officials are also hopeful that the deal will expand the CRO’s small- and mid-sized biotech client base. Small- and mid-sized sponsors have been Charles River’s fastest growing customer segment.

The acquisition is expected to close by the second quarter of this year.