2018 Pipeline Highlights

Each year, new drugs are brought to the market that offer hope to patients who may not have had previous treatment options or who were unresponsive to current therapies. Each drug requires significant preclinical and clinical testing, while some drugs must undergo post-approval testing, as well.

At the National Association of Specialty Pharmacy Annual Meeting and Educational Session, Ray Tancredi, RPh, MBS, CSP, divisional vice president, Specialty Pharmacy Development & Brand Rx/Vaccine Purchasing, Walgreens, discussed the latest FDA approvals and the pipeline for several specialty conditions.

The drug approval process is multi-faceted and involves numerous processes. To ensure that the most promising drugs for the most serious conditions are reviewed quickly for the benefit of the patient, the FDA grants several review designations.

Breakthrough therapy is granted when preclinical evidence suggests that an investigational drug may provide significant improvements over current therapies. Since June 2017, there have been 197 drugs granted breakthrough designations out of 575 requests. Tancredi reported that only 81 products granted breakthrough designation received FDA approval.

Priority review designation is reserved for products that would provide safety and efficacy benefits over current drugs. Under this designation, the FDA has to take action within 6 months. Orphan drug designation is only given to products that treat rare conditions with a patient population of less than 2000.

With the number of high-touch specialty drugs in the pipeline, it is important for specialty pharmacies to keep up with new approvals. In 2017, several specialty drugs received approval, including eteplirsen (Exondys 51) for Duchenne muscular dystrophy, an adalimumab (Humira) biosimilar, and rucaparib (Rubrara) for advanced ovarian cancer.

One of the most groundbreaking approvals of 2017 was ocrelizumab (Ocrevus), which is indicated to treat primary progressive multiple sclerosis (PPMS). Until this approval, there were no treatments for this form of MS, according to the session.

Currently, a majority of drugs treat relapsing remitting (RR) MS, which accounts for 85% of the patient population. Many of these therapies are disease modifying agents administered orally, subcutaneously, or intravenously. Despite the availability of several therapies, none are curative.

Tancredi also highlighted the significance of the May 2017 approval of edaravone (Radicava) for the treatment of amyotrophic lateral sclerosis (ALS). This drug is the first ALS treatment to receive approval since 1992 and works by reducing oxidative stress in the body.

Two drugs for ALS—Mastinib (AB Science) and Tirasemtiv (Cytokinetics)—are expected by 2017 and 2018, respectively, according to the session.

Over the past year, many PD-1, PD-L1, and PD-L2 inhibitors have received approval for new indications, including pembrolizumab (Keytruda), avelumab (Bavencio), and durvalumab (Imfinzi), according to the session. Manufacturers continue to seek new indications for these drugs.

Several oncology products received approval in 2017, bringing therapy to numerous patients with complex and previously untreatable cancers. Tancredi noted the significance of the recent approval of a CAR T cell therapy for cancer and that there is another in the pipeline— axicabtagene ciloleucel—to be reviewed by the FDA by November 29, 2017.

As the specialty landscape continues to change, more disease states are emerging. In the next few years, the FDA will be tasked with reviewing countless applications for specialty drugs. Tancredi gave the audience some highlights for a few disease categories:

Inflammatory disease pipeline:

· Tildrakizumab (Merck, Sun Pharma) for psoriasis. Expected by 2018.

· Gevokizumab (XOMA Corporation) for pyoderma Gangrenosum. Expected by 2018.

Multiple sclerosis pipeline

· Glatiramer acetate—a generic for Copaxone (Sandoz, Mylan, Dr Reddy’s Laboratories, Pfizer) for RRMS. Expected by 2017.

· Nerventra (laquinimod, Active Biotech/Teva) for RRMS and PPMS. Expected by 2018.

· Ozanimod (RPC1063, Celgene) for RRMS. Expected by 2018.

· Cladribine (EMD Serono) for RRMS. Expected by 2018.

· Siponimod (Novartis) for RRMS and secondary progressive MS. Expected by 2019.

· Masitinib (AB Science) for progressive forms of MS. Expected by 2019.

· Ponesimod (Actelion) for RRMS. Expected by 2019.

Oncology pipeline

· Abema (abemaciclib, Eli Lilly) for breast cancer. Expected in 2018.

· Acalabrutinib (AstraZeneca) for chronic lymphocytic leukemia and small lymphocytic lymphoma. Expected in 2018.

· Axicabtagene ciloleucel (Kite Pharma) for refractory non-Hodgkin lymphoma. Expected in 2017.

· Binimetinib (Array BioPharma) for melanoma. Expected in 2018.

· Larotrectinib (Loxo Oncology) Tumors with tropomyosin receptor kinase fusions. Expected in 2018.

· Lutathera (Advanced Accelerator Applications) for neuroendocrine tumors. Expected in 2017.

· MabThera (Roche) for diffuse large B-cell lymphoma. Expected in 2017

· Selumetinib (Array/AstraZeneca) for differentiated thyroid cancer. Expected in 2019.

Atopic dermatitis pipeline

· Tralokinumab (AstraZeneca) expected by 2019+

· Lebrikizumab (Genentech) expected by 2019+

· Otezla (apremilast, Celgene) expected by 2019+

Asthma pipeline

· Benralizumab (AstraZeneca) for eosinophilic asthma. Expected by 2017.

· Tralokinumab (AstraZeneca) for severe asthma. Expected by 2018.

· Lebrikizumab (Genentech) for severe asthma. Expected by 2018.

· Dupixent (dupilumab, Sanofi/Regeneron) for eosinophilic asthma. Expected by 2018.