FDA moves to clear path for drugs aimed at cancer-causing genes

For years, doctors have identified cancers by the affected body part: lung, breast, kidney. Now, in a long-awaited move, U.S. drug regulators will simplify the approval of treatments targeting specific gene mutations that can spur tumors in a variety of organs.

The Food and Drug Administration will soon announce a plan to update agency policies and facilitate the approval of critically needed drugs, including those known as tumor-agnostic therapies, which attack cancer-linked DNA, according to FDA Commissioner Scott Gottlieb.

“This represents some of the biggest opportunities in medicine to treat and cure debilitating and, yes, very costly diseases,” Gottlieb wrote in remarks for his appearance Tuesday before the Senate panel responsible for overseeing the agency’s budget.

The proposal, dubbed the Medical Innovation Access Plan, would answer President Trump’s call to speed drug approvals at what he called the “slow and burdensome” FDA in a February speech before Congress. The agency has been approving cancer drugs more quickly in recent years, but companies would like additional guidance for developing cutting-edge therapies to hasten through the testing phase and into the agency’s review process.

Last month, the FDA approved the first tumor-agnostic drug, expanding the use of Merck’s blockbuster treatment Keytruda to patients with solid tumors that test positive for a certain mutated gene. While the genetic flaw occurs most frequently in colorectal and endometrial cancers, it’s also seen in breast, prostate, bladder and other tumors.

Results from trials of Loxo Oncology’s tumor-agnostic cancer drug earlier this month showed it shrank a variety of tumors in patients with a rare genetic anomaly. One important question is whether the small trial will provide enough information to gain approval, something the FDA could answer as it develops guidance for companies that follow in Loxo’s footsteps.

Within the next six months, the FDA plans to release guidance to help pharmaceutical companies simplify the development of targeted drugs for rare diseases, Gottlieb said.

“We’ll clarify when we may be able to give a broad approval to a cancer drug, in multiple different kinds of molecularly similar cancers, that’s not particular to the tumor being in any one specific tissue or organ,” he said in his prepared remarks.

Since the first full human genome was sequenced in 2003 — providing a complete reading of the body’s instructions for making proteins, tissues and cells — there has been a revolution in understanding what drives tumor growth. Researchers now have a more complete picture of how fused, garbled or incomplete genes can cause the malignant cells to grow at multiple sites in the body. Companies are already working on medicines to attack those flaws, and the lack of a formal approach to testing and treating those medicines would slow their development.

Rick Pazdur, director of the FDA’s Oncology Center of Excellence, predicted at the end of 2015 that a focus on tumor-agnostic treatments would be the next big area of research.

“We’re going to redefine diseases,” he said at the time.

While many drugs target rare diseases, the FDA has a backlog of 200 applications from drugmakers for so-called “orphan drug” designation. Such drugs would treat diseases that affect fewer than 200,000 people in the U.S., and allow drugmakers certain perks, including tax credits for clinical testing.

The FDA will eliminate the backlog in the next three months by placing a team of reviewers solely on the pending applications, Gottlieb said. He also promised that there would never be a backlog again and every orphan drug designation request would get an FDA response within 90 days.

Source: http://www.sfchronicle.com/business/article/FDA-moves-to-clear-path-for-drugs-aimed-at-11234132.php