It’s official.
As the 2017 BIO International Convention went down in San Diego, California, and debate raged about the Better Care Reconciliation Act (BCRA) in Washington D.C., the U.S. FDA approved the 22nd and 23rd new drug entities for the year.
That means 2017 has now topped the final tally from 2016 and even hints at exceeding 2015’s historic high of 45 approvals.
Are they decent drugs? Is there much blockbuster potential? Below are some halftime highlights…
The biggest biopharma winners from the first of half of the year are arguably Regeneron and Sanofi. Together, they codeveloped two new entities; Dupixent (dupilumab) for atopic dermatitis and Kevzara (sarilumab) for rheumatoid arthritis. Back in January, Evaluate Pharma forecast Dupixent could generate as much as $4.5 billion in sales annually by 2022, making it the biggest release of the year.
Another highly anticipated moneymaker is AstraZeneca’s PD-L1 monoclonal antibody durvalumab (Imfinzi), approved on May 1. The drug is now indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma. Evaluate Pharma estimated durvalumab would generate annual sales north of $2 billion by 2022, which would make it the top-selling oncology drug in its review.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
The most controversial approval of the year goes to Emflaza, a corticosteroid indicated for Duchenne muscular dystrophy (DMD). The celebration period was brief for all parties involved, as an $89,000 annual price tag was slapped on the decades-old drug. After much outcry from the media, Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings, (D-Md.) even joined the fray, sending a harshly worded letter to its developer Marathon Pharmaceuticals. Marathon has subsequently offloaded the drug.
Perhaps the biggest surprise in the new drug department came in early May, with the U.S. approval of a Japanese drug for amyotrophic lateral sclerosis (ALS). Dubbed Radicava (edaravone), its developer MT Pharma America managed to get the drug across the line with no U.S. trials. In fact, the company received an inbound call from FDA, which had caught wind of its potential.
“After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,” said Eric Bastings, deputy director of neurology products at the FDA’s Center for Drug Evaluation and Research, in an agency statement.
And finally, a special mention goes to Roche for notching the first-ever drug approval for primary-progressive multiple sclerosis (PPMS), the most severe form of the disease. The therapy, Ocrevus, is a humanized form of an old cancer drug called Rituxan. It is also indicated for the more common relapsing-remitting MS subtype — the makings of a blockbuster franchise.
Looking ahead
Some Superbowl decisions are already lined up for the second half of this year. Novartis is expecting a ruling on its CAR-T immunotherapy in mid-July, the first of its class, and Kite Pharma is close behind.
In Alzheimer’s disease, Axovant is looking to unblind its pivotal trial at the end of September. It may be too late in the year to rush through an FDA approval, but it could give the industry a glimpse into whether the curse of Alzheimer’s drug development has finally lifted.
Let’s hope the new approval streak continues.
Photo: Michail_Petrov-96, Getty Images
