Biologics

CBER Posts Eligibility Criteria for ‘Regenerative Advanced Therapies’

CBER has posted eligibility criteria for qualifying a product as a “regenerative advanced therapy” (RAT) and an accelerated approval pathway as mandated by the 21st Century Cures Act, which was signed into law last month. According to the Center, a product can be eligible for this pathway if:

The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations;
The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition

CBER says that to determine whether a disease or condition is serious or life-threatening and whether a drug is intended to treat a serious disease or condition, stakeholders should refer to an agency guidance on Expedited Programs for Serious Conditions – Drugs and Biologics.

“The request for RAT designation must be made either concurrently with submission of an Investigational New Drug application (IND) or as an amendment to an existing IND,” CBER says. “We will not grant a RAT designation if an IND is on hold or is placed on hold during the RAT designation review. We generally do not expect you to submit primary data (data sets), but your request for designation as a RAT should describe the preliminary clinical evidence. Please include a brief description of any available therapies for the disease or condition, the study design, the population studied, and the endpoint(s) used; and a description of the study results and statistical analyses (e.g., subgroup analyses).”

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome