An Encouraging Snap Shot of New Drug Approvals by FDA in 2017

Over the years, there has been a lot of debate surrounding the new drug approval process, specifically how very few drugs are being approved and the impact it has on patients’ lives. However, both as a keen observer and a critical contributor within the drug development industry, I am encouraged by the number of new drug approvals that were secured in 2017 alongside some notable “firsts”.     

Below is a snap shot of all the approvals that were secured in 2017:

· The FDA approved 46 novel drugs in 2017¹

o To put this in perspective, in the ten-year period from 2008 through 2016, the average was 31 novel drug approvals per year, with only 22 novel drugs approved in 2016. This is a remarkable achievement.

· Of these 46 novel drugs, 15 (33%) were First-in-Class

o Notable novel first-in-class approvals for 2017 include: Dupixent (dupilumab) to treat adults with moderate-to-severe eczema (atopic dermatitis), and Ocrevus (ocrelizumab) to treat adults with relapsing forms of multiple sclerosis and primary progressive multiple sclerosis.

· Of these 46 novel drugs, 18 (39%) were approved to treat rare or “orphan” diseases that affect 200,000 or fewer Americans

o Examples of drugs that advance the care of patients with rare diseases approved in 2017 include:

a) Brineura (cerliponase alfa), a treatment for a specific form of Batten disease, a rare disease that can cause progressive neurological impairments, including seizures, visual problems/blindness, personality and behavior changes, dementia, and loss of the ability to walk, talk, and communicate, and

b) Hemlibra (emicizumab), for the prevention of bleeding or to reduce the frequency of bleeding episodes in patients with hemophilia A who have developed antibodies called Factor VIII inhibitors. This is the first non-blood product approved for this condition.

· Of these 46 novel drugs, 18 (39%) were designated as Fast Track. Fast Track speeds new drug development and review, for instance, by increasing the level of communication between FDA and drug developers, and by enabling FDA to review portions of a drug application ahead of the submission of the complete application.

· Of these 46 novel drugs, 17 (37%) were designated as Breakthrough Therapies. A breakthrough therapy designation includes all the Fast Track program features, as well as more intensive FDA guidance on an efficient drug development program. Breakthrough therapy designation is designed to help shorten the development time of a potential new therapy.

· Of these novel 46 drugs, 28 (61%) were designated Priority Review. A drug receives a Priority Review if FDA determines that the drug could potentially provide a significant advance in medical care. The drug is reviewed within six months instead of the standard 10 months.

· Of these 46 novel drugs, 6 (13%) were under the Accelerated Approval program. The application of accelerated approval brings drugs that can provide important advances to patients sooner than with traditional approvals.

· Of these 46 novel drugs, 28 (61%) were designated in one or more expedited categories of Fast Track, Breakthrough, Priority Review, and/or Accelerated Approval.

· First Cycle Approval

o Of these 46 novel drugs, 39 (85%) were approved on the “first cycle” of review, meaning without requests for additional information that would delay approval and lead to another cycle of review. From 2011 through 2016, FDA approved 204 novel drugs, of which 166 (81%) were approved on the first cycle. The rate for 2017 is consistent with this average. This helps to assure that the application contains the information FDA needs to be able to fully review-and if appropriate-approve an application.

·    Approval in the United States Before Other Countries

o Although regulatory processes differ widely between FDA and those of regulatory agencies in other countries, 36 of the 46 novel drugs approved in 2017 (78%) were approved in the United States before receiving approval in any other country.

·       New uses

o Additionally, there were a total of 14 approvals of already-FDA-approved drugs that were accompanied by new data demonstrating safety and effectiveness of the same product for an additional purpose or for use in a different population of patients. Applications to modify the use of an already-approved drug or to expand its use to other patients are in a category of supplemental applications known as “efficacy supplements.” Notable among them was

§ Keytruda (pembrolizumab), originally approved in 2014 to treat patients with advanced or unresectable melanoma who are no longer responding to other drugs. Throughout 2014 - 2017, it was approved for many new uses to treat patients with various forms of cancer. In May 2017, Keytruda was approved to treat patients whose cancers have a specific genetic feature (biomarker). This was the first time FDA has approved a cancer treatment based on a common biomarker rather than the location in the body where the tumor originated. It was additionally approved in 2017 to include treatment of patients with refractory classical Hodgkin lymphoma, a particularly difficult-to-treat type of Hodgkin lymphoma in which the cancer returns after treatment. It was also approved to treat certain patients with recurrent, locally advanced or metastatic forms of stomach cancer called gastric or gastro-esophageal junction adenocarcinoma.

· New populations

o A total of 3 already approved drugs were approved in 2017 by the FDA for use in expanded population of patients.

· Biosimilars

o The FDA approved five new biosimilars in 2017 compared to 3 in 2016.

Notable “firsts” in 2017:

· Abilify MyCite (aripiprazole tablets with sensor). Abilify was first approved by FDA in 2002, in tablet form, to treat patients with schizophrenia. It was subsequently approved to treat certain patients with bipolar I disorder and as adjunctive treatment of major depressive disorder. In 2017, Abilify MyCite was approved as the first tablet with an electronic sensor allowing patients to track the ingested medication via a smartphone or the cloud. Patients can also provide their caregivers or physician access to their information through a web-based portal.

· Endari (L-glutamine oral powder), the first treatment approved for patients with sickle cell disease in almost 20 years. Prior to this approval, L-glutamine was approved as a prescription drug in 2004 to treat certain patients with short bowel syndrome.

· Juluca (rilpivirine and dolutegravir), a combination of two FDA-approved drugs, each used in the treatment of certain patients with HIV. It was approved in 2017 as a complete regimen for the treatment of certain adults with HIV-1 infection. This is the first complete regimen containing only two HIV-1 drugs and neither is a nucleoside reverse transcriptase inhibitor (NRTI).

These new drug approval numbers for 2017 are certainly impressive, providing all of us engaged in drug development, and the health care industry as a whole, a sense of pride and encouragement regarding our accomplishments. But beyond these numbers, we have also made huge strides in our understanding and application of biomarkers and integrating technology (sensors) in drug development. These innovative approaches along with increased adoption of “seamless clinical trials” wherein combining two or more phases into one adaptive design study, I believe will continue to yield these encouraging numbers of new drug approvals hopefully in this current year and also in the years following.

Uma Arumugam, MD, Director, Clinical R&D, Early Phase Services at ICON Plc.

• https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm