The Milestone

How Rare Disease Shapes Dealmaking

What the Data Tells Us

  1. Rare disease assets now make up about one in three deals, down from about one in two in 2018–19.

    2018–19Last 12 mo
    38%
    of deals signed in the last 12 months, against 48% in 2018–19

  2. Buyers pay about 25% more upfront for rare disease assets than for similar deals.

    RareOthers
    $20M
    median upfront, against $16M for comparable deals

  3. Repurposed drugs show a smaller upfront discount in rare disease.

    RepurposedNew
    53% less
    versus 72% less elsewhere

Rare Disease Share of Dealmaking

Deals per Year

114201814420191452020133202112420221332023122202414020251102026 YTD
  • 48% → 38% share of all deals, 2018–19 against the last 12 months.
  • 1,165 rare disease deals since 2018.
  • 2020 was the busiest full year, with 145 deals.

Rare Disease Pricing Against the Market

2018–Oct 2026

$20M Rare disease $16M Comparable deals
1.3×

Buyers pay about 25% more upfront for rare disease assets than for similar deals: a median of $20M against $16M.

  • At large pharma: $69M against $54M.
  • At smaller buyers: $11M against $10M.
  • Median upfront incl. equity, partnering deals since 2018, matched on stage, buyer size, rights and signing period.

Repurposing in Rare Disease

2018–Oct 2026

Repurposed drugs show a smaller upfront discount in rare disease.

Rare Disease

53% lessupfront vs new drugs
Repurposed$8M
New drugs$17.2M

Other Diseases

72% lessupfront vs new drugs
Repurposed$4M
New drugs$14.5M

Like-for-like median partnering upfronts.

Repurposing accounts for 10% of rare-disease deals and 13% elsewhere; 51% of rare-disease repurposing deals are signed at filing or approval.

Assets Being Licensed

2018–Oct 2026

Rare disease dealsRest of library

Share of each group’s deals in each category. A deal can fall in more than one category. Select a row to see those deals.

Therapeutic area
0%25%50%
Modality
0%25%50%
Stage
0%25%50%

Buyers and Sellers

580 buyers and 834 sellers. Most active buyers: Eli Lilly, Novartis and Merck & Co. 28% of large pharma deals now involve rare disease assets, against 45% in 2018–19.

Most Active Buyers

2018–Oct 2026

EarlierLast 12 months
27
24
21
20
19
18
16
16
14
13

Most Active Sellers

2018–Oct 2026

EarlierLast 12 months
13
13
9
8
6
5
5
5
5
5

Who Buys, by Company Size

2018–Oct 2026

Rare disease dealsRest of library
Large pharma
28%
40%
Mid-size
12%
9%
Small biotech
59%
51%

All Rare Disease Deals

DateDealTypeStageTherapeutic areaIndicationModalityUpfront (incl. equity)Total
22 Oct 2026KG013License—ImmunologyIgA nephropathyAntibody therapeutic—$581M
19 Oct 2026Silence Therapeutics siRNA pipeline and platform, led by D…AcquisitionPhase 2HematologyPolycythemia vera (lead Divesiran programme); br…RNA therapeutic$1.3B$1.3B
13 Oct 2026VIS-102 (HT006.2.2)LicensePhase 1OphthalmologyNeurotrophic keratitisRecombinant protein therapeutic—$111M
5 Oct 2026Lixudebart (ALEF-02)LicensePhase 2ImmunologyANCA-associated vasculitis with rapidly progress…Antibody therapeutic$355M$1.6B
2 Oct 2026ABO2203; options on additional RNA therapeutic programsLicensePhase 1ImmunologyAutoimmune diseasesRNA therapeutic$575M$7.8B
28 Sep 2026SPR2015LicensePreclinicalOncologyKRAS G12D-mutated solid tumorsSmall molecule$400M$2.1B
22 Sep 2026Mantle Therapeutics Friedreich ataxia pipelineAcquisition—NeurologyFriedreich ataxiaSmall molecule$8.3M$20.3M
21 Sep 2026ITM portfolio including ITM-11 (177Lu-edotreotide)AcquisitionPhase 3OncologyGastroenteropancreatic neuroendocrine tumors; Lu…Therapeutic radiopharmaceutical$1.6B$2.4B
17 Sep 2026Marea cardioendocrine antibody portfolio: MAR001/MAR005 an…AcquisitionPhase 2MetabolicSevere hypertriglyceridemia; AcromegalyAntibody therapeutic$352M$352M
15 Sep 2026Unnamed trispecific T-cell engager programme for multiple …Asset acquisitionPreclinicalOncologyMultiple myelomaBispecific/multispecific antibody—$750M

Showing up to 10 example deals for the selected view.

Appendix: How We Count

What counts. Therapeutics deals in the Deal Library tagged as involving a rare disease. A deal not tagged as rare disease is not confirmed as non-rare.

Comparisons. “Rest of library” means every other deal in the library. Partnering covers licenses, options, research collaborations, co-development and asset acquisitions; acquisitions of whole companies are compared separately. Missing figures are never counted as zero. Upfront comparisons are like for like: medians are matched on stage, buyer size, rights and signing period. Trends compare the last 12 months with 2018–19.

Coverage. Library as of 23 Oct 2026.

Info

A Deal Library report on dealmaking in rare diseases.