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Orphans are Getting Strategic

You may have read the new report we recently published on the orphan drug market. Rare diseases and their therapies are a huge topic to cover so inevitably, we couldn’t cover everything in the report. However, that’s what webinars are for! I’m delighted to be taking part in Evaluate’s upcoming webinar which will explore the dynamic landscape of orphan drugs and their shifting dynamics in the pharmaceutical market.

Of course, we’ll discuss the report’s highlights and delve into the financial trajectory of the ten most valuable orphan drugs, projected to collectively generate over $57 billion by 2028. We’ll compare this to the massive $18.5 billion in combined sales that Novo Nordisk recorded in 2023 for Ozempic and Wegovy. This staggering figure represents roughly 32% of the projected 2028 revenue of these top orphan drugs, highlighting the explosive growth and strategic shifts occurring within the industry.

Darzalex has established itself as the frontrunner in the orphan drug market, with sales nearing $9.7 billion and a robust growth rate of 8.1% CAGR. Notably, this rate marks a decline from last year’s projection of 11.7%, indicating a loss of 3.6% in forecasted growth. This downturn highlights emerging market challenges, even for leading products. With its patent set to expire in 2029, Johnson & Johnson faces significant hurdles. Innovatively, they have responded by integrating hyaluronidase into Darzalex formulations, enabling the drug to be administered via injection rather than infusion. This reclassification potentially qualifies it as a new medicine, strategically extending market protection and delaying price negotiation eligibility until possibly 2033 or 2034. This manoeuvre by Johnson & Johnson prompts a crucial question for the industry: Will other companies follow suit with similar strategies to circumvent market challenges?

Our webinar will explore these strategic responses further, examining the effects of patent expirations and the escalating pressures from payers who are now more focused on widespread health issues such as obesity. We will also discuss the critical need for sustained innovation within the orphan drug sector. Continued innovation is essential not only for maintaining commercial viability but also for providing hope and improved treatments to patients suffering from rare diseases, underscoring the sector’s pivotal role in advancing healthcare.

Is the orphan drug market really losing its lustre, or is this just a precursor to a significant strategic pivot that could reinvigorate the sector?

I’ll be part of a panel that features Melanie Senior, author of the Evaluate Orphan Drug report, and Stephanie Léouzon, Managing Director, Biopharma Healthcare at Stifel.

Join us to discover the future of orphan drugs in an evolving healthcare landscape.

Don’t miss this insightful session that promises to enlighten and guide stakeholders across the pharmaceutical industry. I hope to see you there!


Puru Gaur

Senior Commercial Analyst


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