$19.4B of 2025 other-area blockbuster sales lose US exclusivity by 2037, led by Trikafta, Ultomiris, Prolia. These 29 public biotechs have 53 other-area treatments in Phase 3 or filed with the FDA, 26 of them already filed.
2025 sales each company reports from tracked other-area treatments losing US exclusivity by 2037. Big pharma has spent $70.4B on 10 other-area acquisitions since 2023. See the deals →
Gene therapy platform racing three orphan BLAs by 2028; OCU400 Phase 3 readout first proof point, contingent on sustaining $100M
PM359 prime editing therapy for a fatal pediatric immune disease races a BLA filing against a going-concern clock, with survival contingent on capital raising
Gene therapy for blinding retinal disease hits FDA-aligned endpoint in every patient; Phase 3 bet now hinges on whether a small randomized trial replicates it
Four late-stage programs approaching simultaneous approval decisions; encaleret's May 2027 PDUFA with clean Phase 3 data is the first test of whether BridgeBio can convert its pipeline into a multi-franchise rare-disease company
FILSPARI's dual-indication launch drives 71% revenue growth, but scaling into FSGS and defending against emerging IgAN entrants is the proof point
Gene therapy platform with three shots on goal, but AAV2-hAQP1's pivotal readout is the fulcrum: Phase 2 data either opens a clear BLA path or leaves bota-vec's contested filing carrying the entire near-term case
Gene therapy platform with one approval, FDA-aligned Danon pivotal underway, and a PKP2 design update as the next make-or-break gate
LINZESS profits fund a GLP-2 SBS bet; apraglutide Phase 3 readout decides whether Ironwood is a royalty story or a pipeline company
Ciprocopan approved in China, now racing to U.S. IND before China-only data proves insufficient for Western regulators
First agent to beat aflibercept head-to-head in wet AMD, now racing an NDA filing against a runway that stops short of full commercialization
Phase 3 win and rolling NDA filed for a topical rapamycin with no approved rival; approval in H1 2027 is the single gate
Mitapivat reaches a November 1 FDA decision after RISE UP met hemoglobin response but missed significance on pain crises
First-ever STGD1 therapy filing at FDA with a clean Phase 3 win; approval in February 2027 is the single make-or-break event
Axiomer platform's first clinical validation in hand; biliary atresia Phase 2 interim data will determine whether NTCP modulation translates from biomarker to disease benefit
Single-asset BLA filing for a fatal childhood brain disease with no approved treatment; FDA alignment confirmed, but accelerated approval path is the bet
Nafamostat racing a Q4 2026 cash cliff: NEPHRO CRRT readout must deliver before the money runs out
Two CRLs, both manufacturing-only: OLC approval hinges on whether FDA clears the vendor facility
YUVIWEL approved and generating revenue, but a 20% BioMarin royalty burden tests whether achondroplasia economics support the combination therapy ambition
Five Phase 3 wins and a BLA on deck, but going-concern cash burns past the approval decision unless Kodiak raises capital
FAYUVI becomes Ultragenyx’s second approved gene therapy; Aspire failure still weighs on growth
GBS complement inhibitor heads to FDA with striking Phase 3 data; approval verdict determines whether Annexon becomes a commercial company
REDEMPLO approved in five markets; sHTG label expansion with pancreatitis claim vs. approved olezarsen is next value inflection
Outpatient mRNA CAR-T skips lymphodepletion entirely; Phase 3 AURORA tests whether that tolerability comes at the cost of efficacy in MG
EscharEx Phase III interim data arrives as cash runs to Q2 2027, making the readout a survival event, not just a scientific one
Post-bexotegrast pivot bets on ICI-refractory solid tumors; Phase 1b efficacy data will determine if PLN-101095 is real or a single-arm footnote
TARA-002 pursues approval in pediatric LMs and NMIBC; BLA submission and registrational trial data will determine whether multi-indication value materializes
Rare skin disease with no approved treatment, one pivotal study, and $14M to get there
Pivoted FASN inhibitor carries Phase 3 acne data and $258M into a US trial; success or failure hinges on replicating Ascletis results in a Western population
Recorlev-driven commercial momentum funds XP-8121's Phase 3 entry into hypothyroidism, contingent on sustaining revenue growth
Filed treatments are listed first. This shows where late-stage treatments are being developed, not which companies will be acquired. Other covers eye, kidney, liver, blood, bone, growth and rare metabolic diseases. Pipeline data and summaries from Readouts as of 29 September 2026; companies already acquired are left out.
Other areas: Oncology · Immunology · Cardiometabolic · Vaccine · Infectious disease · Neuroscience