Seven new drugs launched this year are projected to achieve more than $1 billion in sales by 2023, according to a new report published by Clarivate Analytics, which owns BioWorld. Abbvie Inc.'s rheumatoid arthritis candidate, upadacitinib, and Novartis AG's Avexis-sourced gene therapy, Zolgensma (onasemnogene abeparvovec), for spinal muscular atrophy (SMA) could lead the way, raking in $2 billion each, while Fibrogen Inc.'s anemia medicine, roxadustat, isn't far behind with anticipated sales of $1.97 billion in 2023. The list, backed by analysis of data from Cortellis, is dominated by immune-related and genetic disorders. Its composition indicates a shift toward targeting niche patient populations bolstered by strong collaborations with big and specialty pharma companies, Clarivate said.
In addition to the aforementioned three, blockbuster drugs expected to launch in 2019 also include medicines for paroxysmal nocturnal hemoglobinuria, psoriasis, peanut allergy and beta-thalassemia. (See chart, below.)
Together, the medicines comprise the Cortellis Drugs to Watch list, a cohort of seven medicines that will face a variety of competitive landscapes.
Among several notable aspects of this year's projected blockbuster crop, only one member, Aimmune Therapeutics Inc.'s peanut allergy immunotherapy, AR-101, is first-in-class – a typical characteristic of blockbusters past. Second, this year's list contains no cancer drugs, something the authors suggested "may highlight the increasing range of new oncology treatments in development, resulting in a smaller potential market share for each one thus lessening the likelihood of any particular drug becoming a blockbuster."
A shift in focus from mass markets to more targeted patient populations also continues to grow, with "the opportunity to develop fast-follower treatments with limited differentiation addressing extremely high prevalence conditions" seemingly "tapped out" the report's authors suggest. Instead, companies are investing more in high-value rare disease indications, especially where incentives put in place by global regulatory bodies have encouraged greater activity. The seven likely blockbusters identified collectively hold nine U.S., four EU and one Japanese orphan drug designations; four FDA breakthrough therapy designations; and two EU PRIME designations.
"It's extremely encouraging to see how these incentives, along with discovery breakthroughs and new R&D approaches, are positively impacting patients in underserved populations. What's more, these novel treatments demonstrate the power of collaboration across the industry, which ultimately accelerates innovation," said Mukhtar Ahmed, president of Life Sciences at Clarivate Analytics.
New this year, the analysis leveraged the Cortellis Analytics Drug Timeline & Success Rates to forecast and understand the probability of drugs considered for the list launching this year, applying statistical modeling and machine learning to forecast the timeline and probability of success for each drug. The authors then leveraged independent analyst and broker reports to understand what the valuation on each program might be, Sarah Hardison, senior analyst for portfolio and market development at Clarivate Analytics, told BioWorld.
Another notable aspect of this year's list is its inclusion of two new gene therapies expected to be launched this year, marking substantial progress on this new technology, Hardison said. They are Zolgensma, which corrects the genetic cause underlying SMA, and Lentiglobin (betibeglogene darolentivec), which corrects the genetic cause of beta-thalassemia, a blood disorder that causes life-threatening anemia.
With a background in immunology, Hardison said she was pleased and excited to see so many immunology drugs expected to launch this year. "Underpinning what we predict will come out this year is our ever-increasing understanding of the basic biology of immunity. I think that R&D is doing a very good job of keeping up with what's happening in novel discovery biology and applying that in a therapeutic setting."
Not shy about taking a clear look at its recent track record, the Cortellis analysts behind the report also reviewed the current status of 12 drugs that were expected to enter the market in 2018 and were forecast to achieve blockbuster annual sales of $1 billion or more by 2022. "As predicted, all of those drugs have entered the market. Of the drugs for which sales forecasts are currently available, all but two are still expected to achieve blockbuster status by 2022," they said.
Making clear just how variable each year's blockbuster crop can be, two mass-market drugs on the 2018 list are dominating so far: the HIV drug Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide fumarate, Gilead Sciences Inc.) has forecast 2022 sales of $6.03 billion, while Ozempic (semaglutide, Novo Nordisk A/S) is on track for forecast 2022 sales of $3.62 billion.