Breakthrough “one and done” treatments such as Novartis’ Zolgensma and bluebird bio’s Lentiglobin will contribute to a 12% compound annual growth rate (CAGR) for orphan drugs through to 2024, according to a report by Evaluate Pharma.
The pharma analysis group says these products, in addition to recently marketed cell and gene therapies such as Kite’s CAR-T Yescarta and Spark’s inherited retinal dystrophy drug, will cause the leap.
Non-orphan products are also expected to grow by 2024, but by 6% each year.
“Seven of the top 20 R&D orphan products by net present value are either a cell or a gene therapy,” said report author Karen Pomeranz.
“However, we may not see the full market impact of these drugs by 2024, which means that the long-term growth and patient benefits could be even more pronounced.”
The report also revealed other interesting developments, including Celgene toppling Novartis as the world’s biggest orphan drug developer as its multiple myeloma therapy Revlimid is predicted to be the leading orphan drug product in 2024, and will help bring in total orphan drug sales of $13.7bn for the company.
AbbVie, Johnson & Johnson and Novartis will closely follow Celgene, and “all of the top four companies will garner the majority of their 2024 orphan drug sales from products used to treat haematological malignancies”.
Revlimid generated sales of $2.6bn in Q1, up from $2.2bn sales this time last year, but will see generic erosion by 2024.
Big Pharma tapping smaller companies for its orphan drugs will continue to dominate M&A strategies, which has been a trend of late with the likes of Roche’s $4.8bn acquisition of Spark and along with it Luxturna, and Novartis’ $8.7bn buyout of AveXis and its spinal muscular atrophy(SMA) candidate, Zolgensma.
“Growth rates approximately double those of the overall prescription drugs market and the mean cost per patient per year of the top 100 orphan products in the US hitting $150,854 in 2018 versus $33,654 for a non-orphan drug, explain much of the allure,” says the report.
However this bonanza has generated scrutiny of industry prices, and the very favourable incentives for orphan drugs. The report warns the promise of innovative products such as Zolgensma and Lentiglobin “will only become reality if the innovation of drug companies is matched by innovation in the drug pricing and reimbursement systems”.
The recent explosion of cell and gene therapies has come with eye-watering prices. Luxturna’s current list price is $425,000 per eye, which ICER says is far too high, and the independent drug pricing watchdog has already urged Novartis to develop a fair pricing strategy for Zolgensma.