Earlier this year, we released the Cortellis Drugs to Watch 2019 report, which highlighted seven drugs expected to launch in 2019 and become blockbusters in 2023. We have subsequently reported on three of those drugs that have now gained approval, including Zolgensma which entered the market as the most expensive drug ever launched. Since that time, Rinvoq (upadacitinib) has been approved and launched in the U.S.; Ultomiris has been launched in the EU and Japan and received additional U.S. approval for a second indication; and Evrenzo (roxadustat) has been approved in Japan and its label expanded in China. Meanwhile, Palforzia (AR-101) has faced some delays and its regulatory filing is still under FDA evaluation. 1-8
Rinvoq launches in the U.S. and gets positive CHMP opinion
Rinvoq, AbbVie’s oral JAK1-selective inhibitor, received U.S. approval in rheumatoid arthritis (RA) in August 2019, launching shortly after.1,2 Rinvoq has demonstrated remarkable efficacy in a wide range of RA settings, achieving all primary and ranked secondary endpoints in the phase III SELECT program. By leveraging these data, in particular, superior efficacy to best-in-class Humira, Rinvoq stands to gain a substantial share of the crowded RA market.1
The product is also being reviewed for approval in Japan and the EU, with the EMA’s Committee for Medicinal Products for Human Use (CHMP) adopting a positive opinion in October 2019.9 The Cortellis Analytics – Drug Timeline & Success Rates tool forecasts EU approval in December 2019 and Japan approval in June 2020, with a likelihood of success of 95% for both regions.
Ultomiris now available in multiple markets
Alexion Pharmaceuticals received Japanese and EU approval for its complement inhibitor Ultomiris in June and July 2019, respectively, followed by launches in those regions.3, 4 Ultomiris is a next-generation follow-on to Alexion’s market-leading Soliris for paroxysmal nocturnal hemoglobinuria, a rare and progressive blood disorder that is fatal in approximately 50% of cases. Ultomiris has the potential to become the new standard of care over Soliris by leveraging its non-inferiority and greatly improved dosing convenience (every eight weeks versus every two weeks).10
Ultomiris also received additional U.S. approval in October 2019 for atypical hemolytic uremic syndrome (aHUS), an ultra-rare disease that causes progressive kidney failure and can lead to death. Ultomiris is also set to replace Soliris in this setting.5 Filings for aHUS in the EU and Japan are also being reviewed, with the Cortellis Drug Timeline & Success Rates tool forecasting a 95% likelihood of approval in mid-2020 for both regions.
Evrenzo gets approval in Japan for anemia with CKD and secures label expansion China
AstraZeneca/FibroGen/Astellas’ Evrenzo, a first-in-class oral HIF-PH inhibitor, received Japanese approval in September 2019 for anemia in chronic kidney disease (CKD) patients on dialysis.6 In Mainland China, a label expansion to include non-dialysis-dependent patients was approved in August 2019.7
In the U.S. and EU, submissions for both dialysis-dependent and dialysis-independent patients are being prepared.11 Evrenzo showed no increased risk in major cardiovascular events compared with epoetin alfa in a pooled safety analysis required by the FDA and the EMA.12 The Cortellis Drug Timeline & Success Rates tool forecasts a 90% chance of approval for both regions.
Palforzia moves closer to approval despite delays
Palforzia, Aimmune Therapeutics’ oral desensitization immunotherapy for peanut allergy, is under review for U.S. approval. Two setbacks have delayed the drug’s potential market entry: The U.S. government shutdown in December 2018/January 2019 and the FDA decision to consider Palforzia an allergenic product candidate and therefore not eligible for expedited review associated with its Fast Track and Breakthrough Therapy designations.8 Despite discussions with the FDA, the filing was given a 12-month target period with an action date expected in late January 2020. However, Palforzia is now closer to becoming the first FDA-approved therapy for peanut allergy, following a positive recommendation from the Allergenic Products Advisory Committee (APAC) in September 2019.13
In the EU, a filing was submitted in June 2019 and is currently under review.14 The Cortellis Drug Timeline & Success rates tool forecasts a 95% probability of approval, in October 2020.
Our analysis in the Cortellis Drugs to Watch 2019 report identified seven drugs expected to launch this year with sales forecasts of $1 billion or more by 2023. The four products discussed above provide a fascinating picture of the latest trends in the pharmaceutical industry, giving examples of drugs successfully entering highly crowded markets, of new treatments being approved for rare diseases, and of cutting-edge science being used to develop products capable of monopolizing whole new markets.
Click here to read the full report: Cortellis Drugs to Watch.
- Form 8-K – Aimmune Therapeutics Inc (16/01/19)
- Form 8-K – Alexion Pharmaceuticals Inc (21/12/18)