Success Rate for Biopharma Companies Rising as FDA Approves Near-Record Number of NMEs

It was a busy December for the FDA as it gave the green light to five more new molecular entities (NMEs), boosting the number of NMEs and new therapeutic biological products approved by the U.S. agency in 2015 to 45, an almost 10 percent increase over the 41 NMEs that were approved last year. (See BioWorld Insight, Dec. 29, 2014.).

The total ranks as the second highest number of approvals just behind the 1996 total when 53 new medicines were approved.

The most recent decision was the Dec. 22 approval of Astrazeneca plc’s Zurampic (lesinurad), a week earlier than expected. The new drug is indicated for treating high levels of uric acid in the blood associated with gout when given together with xanthine oxidase inhibitors (XOIs). Combining Zurampic with XOIs such as allopurinol or Uloric (febuxostat, Takeda Pharmaceuticals America Inc.), medicines that comprise the current standard of care, will provide a dual mechanism of action to increase excretion and decrease production of uric acid, enabling more patients with inadequately controlled gout to achieve target treatment goals, the company said. (See BioWorld Today, Dec. 24, 2015.)

Astrazeneca acquired Zurampic through its purchase of Ardea Biosciences Inc. for $1.26 billion in 2012.

The success rate of biopharmaceutical companies getting products over the regulatory goal line has been steadily improving. In fact, over a span of 12 years the annual average of NMEs approved by the FDA has reached 29.

PRIORITY REVIEW

In this year`s crop of new medicines (See New Molecular Entity and New Therapeutic Biological Product Approvals for 2015, below) the agency reported that over half were approved under priority review and 19 of them were granted orphan drug designations.

Among this group was South San Francisco-based Genentech Inc.’s Alecensa (alectinib) for treating anaplastic lymphoma kinase (ALK)-positive non-small-cell lung cancer (NSCLC) patients who have progressed or are intolerant to Xalkori (crizotinib). The FDA granted accelerated approval to the breakthrough therapy-designated drug well ahead of the March 4, 2016, PDUFA date.

Early approval was based on overall response data from phase II studies in ALK-positive NSCLC patients who had progressed on Pfizer Inc.’s Xalkori, including data showing even higher response rates in a subset of patients whose tumors had spread to the brain or the central nervous system (CNS). Data from the 87-patient NP28761 trial, presented at the American Society of Clinical Oncology (ASCO) meeting earlier this year, showed that Alecensa shrank tumors in 47.8 percent of patients, as measured by RECIST criteria. Patients whose disease had spread to the CNS had an overall response rate of 68.8 percent.

Study NP28763, which enrolled 138 patients, showed that Alecensa shrank tumors 40.8 percent, with the CNS subset showing an overall response rate of 57.1 percent.

Alecensa also received orphan drug designation, which provides incentives such as tax credits, user fee waivers and eligibility for exclusivity to assist and encourage the development of drugs for rare diseases.

Priority review was granted to Eisai Co. Ltd.’s Lenvima (lenvatinib), which the agency approved to treat patients with progressive, differentiated thyroid cancer (DTC) whose disease progressed despite receiving radioactive iodine therapy (radioactive iodine refractory disease). The drug also received orphan product designation. Lenvima was approved approximately two months ahead of the prescription drug user fee goal date, which was among the 95 percent of approvals in 2015 that the FDA said met or improved upon their PDUFA goal dates for the approval review cycle.

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