Stephen Hahn Sworn in as FDA Commissioner

Stephen Hahn was sworn in as the FDA’s 24th commissioner by HHS Secretary Alex Azar on Dec. 17.

During his confirmation hearing, the former head of the MD Anderson Cancer Center voiced his support for faster approval of generics and vowed to ramp up drug approvals and innovation. He also suggested greenlighting more non-opioid treatments for pain as a way to help tackle the opioid epidemic.

Unlike many of his predecessors, Hahn lacks direct agency experience. But five former commissioners wrote a letter to Congress in support of his confirmation. The former agency heads noted his seven-year tenure as the National Cancer Institute’s chief of prostate cancer and far-reaching clinical research experience in the public sector.

“I am a scientist and medical doctor and believe strongly in disciplined research, adhering to sound data and upholding the law,” Hahn said, in his first email to agency staff on Dec. 18.

Trump Signs Budget Package with Increased FDA Funding

President Trump signed a fiscal 2020 federal budget package into law Friday that increases funding for the FDA for the third time in three years.

The budget includes $3.16 billion in appropriations for the agency, an increase of 3 percent on the fiscal 2019 allocation. Including user fees, the FDA’s total fiscal 2020 budget is $5.8 billion. Following two previous years of significant increases, the agency is no longer “running on fumes,” according to the Alliance for a Stronger FDA.

The budget expands funding for human drugs by $20 million to $683 million and for biologics by $12 million to $252 million. It also includes additional funding for advanced manufacturing, opioids and rare cancer therapies.

The agency also received additional funding for the 21st Century Cures Act of $5 million to $75 million.

One notable provision expands the definition of biosimilars to include “chemically synthesized peptides” — a category that includes every type of insulin currently on the market. The expanded definition could open up the market for more insulin competitors and lower the drug’s price.

The provision “provides the potential for chemically synthesized follow-on insulins and other protein products to come to market through more efficient abbreviated pathways regardless of how they are manufactured,” an FDA spokesperson told CenterWatch Weekly.

The federal spending package passed the House 297-120 and the Senate 71-23 last week in an end-of-year scramble to meet a Dec. 20 deadline.

Sarepta Gets Approval on Rejected DMD Drug

In an about-face, the FDA has reversed its decision against Sarepta’s formerly rejected Duchenne muscular dystrophy (DMD) drug and granted accelerated approval for the therapy.

The drug, Vyondys 53, targets a specific DNA error occurring in only 8 percent of all Duchenne patients, and the FDA based its reversal on a small increase in an important muscle protein called dystrophin that is normally missing in children with the disease.

The FDA previously rejected Vyondys 53 in August over the risk of infections related to infusion ports and kidney toxicity in animal studies.

Sarepta also ran into hang-ups with adverse event reporting during one of its trials for DMD. But Sarepta contested the FDA’s adverse event reporting submission on a patient enrolled in its microdystrophin trial for DMD, saying that it had not reported any serious adverse events. The company later called the submission “erroneous” and continued the trial (CenterWatch Weekly, Nov. 15, 2019).

Sarepta has not yet proven that Vyondys 53 can improve muscle function or slow the progression of the disease.

Algorithm Accurately Predicts Odds of Drug Approval

Researchers have developed a machine-learning algorithm that can predict a drug’s chances of regulatory approval, according to a recently published study in the Journal of Clinical Oncology Clinical Cancer Informatics.

Because oncology drugs are experiencing a decreasing rate of approval, researchers wanted to develop a tool that would predict the odds of approval for drugs in early phase trials. The result was RESOLVED2, a machine-learning algorithm whose key drivers are phase 1 clinical data, general pharmacologic data and the FDA’s approval timeline for drugs approved from 1972 to 2017.

Using the algorithm, scientists at the Gustave Roussy Institute, in Villejuif, France, and Paris-Sud University used data on an already approved drug to predict that it was 16 times more likely to be approved by the FDA than a nonapproved drug.

Six years of testing of the algorithm showed a high success rate: 73 percent of drugs predicted to be approved had been approved and 92 percent of drugs predicted to be nonapproved were still not approved.

Novartis Hits a Wall in Phase 3 with Drug for Asthma

Novartis announced that it is dropping two phase 3 trials of an asthma drug, fevipiprant (QAW039).

Even with the late phase failure of fevipiprant, Novartis is not giving up on its respiratory drug pipeline as it continues to invest in other products, such as Xolair (omalizumab) and other phase 3 candidates, such as QVM149 and QMF149.

Janssen, PRA Launch All-Virtual Cardiac Trial

Janssen and PRA Health Sciences are collaborating on one of the first fully virtual phase 3 trials for heart failure.

The trial, Canagliflozin: Impact on Health Status, Quality of Life and Functional Status in Heart Failure (CHIEF-HF), will evaluate the impact of Janssen’s Invokana (canagliflozin) on the quality of life improvement in patients with heart failure and type 2 diabetes. The trial will use wearable technology developed by PRA Health Sciences.