Andrzej Rostek
Orphan drugs — medicines to treat rare diseases — from Johnson & Johnson (NYSE:JNJ), Roche (OTCQX:RHHBY), and AstraZeneca (NASDAQ:AZN) will allow these companies to reign as the top 3 orphan drugmakers, by sales, in 2028.
A new report from Evaluate found that Johnson & Johnson will be the top orphan drug pharma, bringing in $30.6B from that class alone. Roche is slated to bring in $16.8B in orphan drug sales, followed closely behind by AstraZeneca at $16.4B.
Rounding out the top 5 are Novartis (NVS) at $13.9B and Vertex Pharmaceuticals (NASDAQ:VRTX) at $12.4B.
The report notes that in 2028, Amgen (AMGN) will have replaced AbbVie (ABBV) in the top 10 thanks to the former’s acquisition of Horizon Therapeutics which closed in October 2023. That deal gave Amgen Tepezza (teprotumumab) for thyroid eye disease, Krystexxa (pegloticase) for gout, and Uplinza (inebilizumab) for neuromyelitis optica spectrum disorders, in addition to Horizon’s pipeline.
For background, the FDA says that a rare disease is one that affects less than 200,000 people in the U.S., adding that more than 7,000 such diseases impact 30M people in the country.
Evaluate finds that the top 3 orphan drugs in 2028 will be Johnson & Johnson’s (JNJ) multiple myeloma therapy Darzalex (daratumumab), Vertex’s (VRTX) cystic fibrosis treatment Trikafta (elexacaftor/tezacaftor/ivacaftor), and Roche’s (OTCQX:RHHBY) hemophilia A therapy Hemlibra (emicizumab).
In 2028, Darzalex is expected to bring in $17B worldwide; Trikafta, $8.8B; and Hemlibra, $6.2B. Rounding out the top 5 are AstraZeneca’s (AZN) Ultomiris (ravvulizumab) for paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and several other rare disorders, and J&J’s CAR-T immunotherapy Carvykti (ciltacabtagene autoleucel) for multiple myeloma. Ultomiris and Carvykti are projected to bring in, respectively, $5.2B and $4.4B.
While oncology indications currently dominate orphan disease therapies — and will continue to do so in 2028 — the pharma commercial intelligence, news, and analysis firm projects that its dominance will ebb slightly towards the end of the decade.
Oncology presently accounts for 44% of orphan disease treatments. That figure is expected to fall to 42% in 2028. Disease areas such as central nervous system, musculoskeletal, and immunomodulators are expected to grow, from 9%, 4%, and 4%, respectively, to 11%, 6%, and 7%.
“Oncology has come off the boil as precision medicines tackling ever-narrower genetic mutations run out of road commercially (druggable ‘pan-cancer’ mutations are elusive), and as CAR-T therapies continue to face challenging administration logistics,” Evaluate notes.
“CNS orphan drug sales will more than double between 2023 and 2028, by which time they’ll make up over 10% of orphan sales by value” Evaluate adds. “This reflects broader industry interest in this once-neglected area.”
