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Beacon Breaking News – Carfilzomib To Get Standard, Not Priority, FDA Review

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Published: Dec 11, 2011 9:29 pm

Onyx Pharmaceuticals, the U.S. company developing carfilzomib as a potential new treatment for multiple myeloma, announced earlier today that the U.S. Food and Drug Administration (FDA) has granted “standard review designation” to the company’s New Drug Applica­tion for carfilzomib.

This means that the FDA expects to make a decision by July 27, 2012, on Onyx’s application to market carfilzomib (Kyprolis) in the United States.

The announcement is a disappointment for Onyx, which had hoped to receive a so-called “priority review” of the carfilzomib application.

The target FDA review time for applications granted a priority review is four months shorter than for drugs with a standard review designation.

If the FDA makes a positive decision regarding carfilzomib’s new drug application by the end of next July, Onyx will be allowed to start marketing the drug in the United States as a new treatment for relapsed and refractory myeloma.

A negative decision by the FDA would delay the launch of carfilzomib until Onyx is able to address any questions or concerns raised by the FDA.

In its press release about the FDA standard review designation, Onyx explained that the FDA decided on a standard review for at least two reasons.

First, the FDA said that its advisory committee of outside oncology experts increasingly is recommending that the FDA not grant priority review unless a new drug application is based on a Phase 3 clinical trial.

This is not the case with the carfilzomib application, which is based on data from a Phase 2 trial.  Phase 2 trials are smaller than Phase 3 clinical trials, and they are often not as scientifically rigorous as Phase 3 trials.

Second, Onyx said that “the FDA outlined potential review issues including whether the application is sufficient to support an FDA conclusion that the data provided within the [New Drug Application] meets accelerated approval criteria and whether the benefit and risk are appropriately balanced, given that the application is based on a single-arm study.”

The statement that “the FDA outlined potential review issues” will be of concern to those hoping for an FDA approval of carfilzomib by next July.

The statement could be interpreted as meaning the FDA has broader concerns about the carfilzomib application — not just concerns about granting the application priority review.

Onyx has scheduled a conference call for Monday morning at 8:00 a.m. Eastern Time to discuss the FDA decision.

Until carfilzomib receives FDA approval, the drug is available through Onyx’s expanded access program for relapsed and refractory myeloma patients in the U.S. who do not have any other treatment options.

Carfilzomib works similarly to the existing multiple myeloma treatment Velcade (bortezomib) by preventing the breakdown of protein in cancer cells, triggering their death.

Along with pomalidomide – a chemical relative of multiple myeloma drugs Revlimid and thalidomide (Thalomid) – carfilzomib is considered one of the most promising new myeloma treatments that could be approved by the FDA in the next few years.

Photo by gtrwndr87 on Flickr – some rights reserved.
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