Angus Liu | Fiercepharma
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.
The top 10 drug launches of
2019
