M&A activity in orphan and rare diseases

A Feature on the deals being done in a lively sub-sector of the pharmaceutical industry.

In August, the US Food and Drug Administration (FDA) gave the nod to bluebird bio's (Nasdaq: BLUE) gene therapy Zynteglo (betibeglogene autotemcel), for the treatment of beta thalassemia; this month the agency approved the company’s second gene therapy Skysona (eli-cel), for early, active cerebral adrenoleukodystrophy (CALD). Investment in rare diseases continues to attract pharma and investor attention and future growth will be driven by scientific and analytical advances and changes in the regulatory and reimbursement environment to enable more rapid access to innovative treatments.

Reseach from Golden Wire indicates that the global orphan diseases market size is projected to reach $547.3  billion by 2030, from $119.6 billion in 2021, and there are still significant opportunities to capitalize on future growth in this space given that more than 90% of identified rare diseases lack an approved treatment option.