Expedited Approvals and Orphan Drugs Dominated 2015 Novel New Therapies

With a flurry of activity in the last two months of the year, FDA approved 45 novel new drugs in 2015 (1), the highest number of approvals since 1996 and second-highest ever (2). “But far more important than quantity is quality-and the valuable new roles many of these drugs can serve in advancing medical care and the health of patients,” said John Jenkins, director of FDA’s Office of New Drugs in the Center for Drug Evaluation and Research (CDER) (3).

The agency approved 87% of the applications on the first cycle of review and met or exceeded the goal dates under the Prescription Drug User Fee Act for all but two of novel new drugs approved. FDA’s expedited pathways for drug approval again proved successful in 2015:

• Fourteen drugs were designated by CDER as Fast Track, with the potential to address unmet medical needs.

• Ten drugs were designated as Breakthrough therapies, with preliminary clinical evidence demonstrating that the drug may result in substantial improvement on at least one clinically significant endpoint over other available therapies.

• Twenty-four drugs were designated Priority Review, in which CDER determined that the drug could potentially provide a significant advance in medical care and set a target to review the drug within six months instead of the standard 10 months.

• Six drugs received early approval under the Accelerated Approval program, which allows early approval of a drug for a serious or life-threatening illness that offers a benefit over current treatments.

• In addition, 16 new drugs (36% of those approved) were identified by CDER as first-in-class, with mechanisms of action different from those of existing therapies, and 21 new drugs (47% of those approved) were approved to treat rare or “orphan” diseases that affect 200,000 or fewer Americans.

Novel new drugs approved in 2016
In addition to the 30 novel new therapies reported previously (4), the following new drugs were approved by FDA between Nov. 1–Dec. 31, 2015:

• Zurampic (lesinurad) from Ardea Biosciences to treat high blood uric acid levels associated with gout

• Uptravi (selexipag) from Actelion Pharmaceuticals to treat pulmonary arterial hypertension

• Bridion (sugammadex) from Organon USA to reverse effects of neuromuscular blocking drugs used during surgery

• Alecensa (alectinib) from Alexion Pharmaceuticals to treat ALK-positive lung cancer

• Kanuma (sebelipase alfa) from Alexion Pharmaceuticals to treat patients with lysosomal acid lipase (LAL) deficiency

• Empliciti (elotuzumab) from Bristol-Myers Squibb to treat people with multiple myeloma who have received one to three prior medications

• Portrazza (necitumumab) from Eli Lilly to treat patients with advanced (metastatic) squamous non-small cell lung cancer who have not previously received medication specifically for treating their advanced lung cancer

• Ninlaro (ixazomib) from Millennium Pharmaceuticals to treat people with multiple myeloma who have received at least one prior therapy

• Darzalex (daratumumab) from Janssen Biotech to treat patients with multiple myeloma who have received at least three prior treatments

• Tagrisso (osimertinib) from AstraZeneca to treat certain patients with non-small cell lung cancer

• Cotellic (cobimetinib) from Genentech to be used in combination with vemurafenib to treat advanced melanoma

• Genvoya, a fixed-dose combination tablet containing elvitegravir, cobicistat, emtricitabine, and tenofovir alafenamide, from Gilead Sciences for use as a complete regimen for the treatment of HIV-1 infection in adults and pediatric patients 12 years of age and older

• Nucala (mepolizumab) from GlaxoSmithKline for use with other asthma medicines for the maintenance treatment of asthma in patients age 12 years and older.

References
1. FDA, Novel New Drugs Summary 2015, online accessed Jan. 5, 2105.
2. FDA, Summary of NDA Approvals & Receipts, 1938 to the present, online  accessed Jan. 5, 2015.
3. J. Jenkins, 2015: Another Strong Year for Patients in Need of New Drug Therapies, online, accessed Jan. 5, 2015.
4. C. Challener, Pharmaceutical Technology 39 (12) 2015.