Cancer is probably the most sought-after indication in the biopharma world, but it’s missing from 2019's class of top drug launches. Instead, treatments in immunology and rare genetic diseases have grabbed the limelight.
We considered two EvaluatePharma projections in January and February to rank this year’s top 10 drug launches by 2024 sales. At the top of the list is Alexion’s Soliris follow-up, Ultomiris, which nabbed an FDA nod at the end of 2018, two months ahead of its scheduled decision date.
As part of CEO Ludwig Hantson’s plan for Alexion to pivot away from ultrarare diseases and reach more patients, the biotech has priced Ultomiris at a 10% discount to Soliris and is aiming to convert 70% of Soliris patients to the improved therapy. Paroxysmal nocturnal hemoglobinuria (PNH), the current condition Ultomiris is approved in, is a rare disease that involves the immune system.
Notably, two gene therapies are included here: Novartis’ spinal muscular atrophy treatment Zolgensma—which could challenge Biogen’s SMA treatment Spinraza—and Bluebird Bio’s Zynteglo for beta-thalassemia. Gene therapies have attracted much interest and controversy alike. As one-time treatments, they come with big efficacy promises, but pricing of these two drugs has been closely watched, and both companies are offering up novel models such as installments paid over several years.
Novartis boasts three drugs on our list, the most among all companies, putting CEO Vas Narasimhan’s innovative medicine-focused strategy to an early test. Besides Zolgensma, the Swiss drugmaker has brolucizumab, a wet age-related macular degeneration therapy that will go up against Bayer and Regeneron’s Eylea and its Roche-partnered Lucentis. In head-to-head studies, brolucizumab topped Eylea in some secondary endpoints.
Novartis' Mayzent, the first-ever oral drug for secondary progressive multiple sclerosis (SPMS), was approved by the FDA in March. Up to 80% of patients with relapsing remitting MS develop SPMS, but the challenge lies in helping doctors identify those patients. The company is focusing on educating patients and physicians to help raise awareness of this stage of the disease and its symptoms.
In the immunology realm, AbbVie has two potential blockbusters on this list, Skyrizi and upadacitinib. The company is trying to diversify its portfolio as its megablockbuster in the field, Humira, starts to decline amid ex-U.S. biosimilar competition. In a recent note to investors, Credit Suisse analysts predicted that every submarket in the anti-inflammation space, be it arthritis, asthma, inflammatory bowel disease or multiple sclerosis, has room for billions in growth.
Other contenders here include Aimmune Therapeutics’ AR101, which aims to be the first drug that reduces children’s allergic reactions to accidental peanut exposure. It hit a speed bump when its application for FDA approval ran into the government shutdown in December but is now under review—and Aimmune's in talks with the agency about speeding up its approval after that delay.
Two drugs facing new questions as they ramp up round out the list: anemia drug roxadustat, which FibroGen is developing in partnership with AstraZeneca and Astellas, actually has already won its first approval—in China. But a pooled analysis of phase 3 safety on the drug, meant for chronic kidney disease patients, recently sent some confusing signals to investors.
Meanwhile, Johnson & Johnson’s ketamine-like depression nasal spray Spravato is also nothing short of controversial, both around its price tag and safety risks.
As we were publishing this report, Bluebird disclosed a delay in Zynteglo's launch. But we decided to keep it on the list because the biotech is planning to enroll patients within this year and treat the first commercial patients in early 2020.
The 2019 class of top drug launches shows the booming of expensive orphan drugs that are based on novel technologies, as well as the undying enthusiasm about the growing immunology market whose opportunity is evident in the world's best-selling drug Humira. As always, we welcome your thoughts and comments. — Angus Liu (email) (Twitter)