$12.9B of 2025 neuroscience blockbuster sales lose US exclusivity by 2037, led by Ocrevus, Kesimpta. These 42 public biotechs have 60 neuroscience treatments in Phase 3 or filed with the FDA, 29 of them already filed.
2025 sales each company reports from tracked neuroscience treatments losing US exclusivity by 2037. Big pharma has spent $59.8B on 7 neuroscience acquisitions since 2023. See the deals →
Ionis has three approved drugs and two fresh Phase 3 wins, but the hepatitis B functional-cure PDUFA is the single event that rerates the stock
Two NDAs at FDA simultaneously, both past mid-cycle with no major concerns flagged; approval of either or both validates Praxis's precision epilepsy engine
Phase 3 data in hand, advisory committee voted 3-9 against; FDA's Aug 22 PDUFA on amended filing is the only question that matters
ENTR-601-44 hit a functional endpoint at first dose; higher-dose readouts and a runway expiring Q3 2027 now determine whether this DMD platform survives to registrational data
NRX-100 clears first-cycle FDA review with no drug deficiencies; approvability via Real World Evidence is the unresolved bet
Azetukalner NDA submitted on strong FOS data, while a voluntary psychiatry enrollment pause adds safety uncertainty
Biohaven pivots to BHV-1300 in Graves' disease after troriluzole CRL and OCD failure, with a $795M Kv7 licensing deal buying time for a 2027 Phase 3 readout
GTx-104 cleared Phase 3 and FDA's clinical bar; a manufacturing-only CRL stands between Grace and approval if remediation completes before cash runs out
Flush with $1.5B from selling sevasemten, Edgewise bets the new company on whether EDG-7500 can carve space from aficamten in a crowded HCM market
Cytisinicline's Q4 resubmission must clear the CRL's manufacturing and labeling issues before a potential 2027 launch
EU rejection forces blarcamesine back to Phase 3 in Alzheimer's; Rett syndrome and $118M cash keep the story alive pending FDA alignment
GBS complement inhibitor heads to FDA with striking Phase 3 data; approval verdict determines whether Annexon becomes a commercial company
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
CMC-only CRL clears the clinical bar but leaves approval timing uncertain; resubmission success determines whether the trimodal dexmethylphenidate ADHD bet pays off
FDA-confirmed accelerated approval path for ALS hinges on NDA acceptance and whether NfL qualifies as a surrogate; runway expires the same quarter the filing lands
Two positive Phase 3 trials, NDA filed, CNPV in hand; approval clears the path; DEA rescheduling determines whether launch actually happens
Specialty ophthalmic rollup with Phase 3-proven sedation drug racing a widening cash burn toward an NDA filing
Rolling miv-cel BLA underway in stiff person syndrome; FDA's view of single-arm data remains decisive
BLA filed on novel surrogate endpoint for fatal orphan disease; FDA acceptance of skin frataxin as the surrogate determines whether approval follows
Ryoncil's $115M launch year funds the rexlemestrocel-L CLBP Phase 3 bet; the readout determines whether Mesoblast is a commercial platform or a one-product story
Quince shed its founding A-T program and rebuilt entirely around inhaled rapamycin; the BOS readout is the first test of whether LAM-001 works in a controlled setting
Sarepta's $1B+ revenue base hinges on FDA granting traditional approval for two exon-skipping drugs whose confirmatory trial missed its primary endpoint
BRIUMVI nearing $1B annual revenue with SC formulation and indication expansions as the growth ceiling test
BLA filed for first Huntington's gene therapy, but 4-year data missed primary endpoint; FDA verdict on external-control adequacy is all that matters
Three consecutive Phase 3 wins with lysergide ODT across GAD and MDD; approval of a novel psychedelic therapy hinges on FDA's regulatory posture at the NDA
NLRP3 inhibitor moving into CV risk patients; thesis stands or falls on whether hsCRP inhibition in diseased subjects arrives before Q4 2027 runway
FDA-aligned Phase 3 design in hand for an MSA drug with no approved rivals, but Phase 3 launch hinges on closing a partnership before Q3 2027 cash runs out
DLB drug with FDA-aligned Phase 3 path but no partner yet; post-hoc subgroup data must hold up in a registrational trial CervoMed cannot fund alone
Dual-BTD psilocybin analog in two Phase 3 pivotals for MDD, but a $42M/quarter burn means survival to NDA hinges on APPROACH data and follow-on financing
GeneTAC platform's first clinical signal in Friedreich ataxia hinges on whether 12-week frataxin data can support a registrational path
CRISPR's first potential approval races a live clinical hold: lonvo-z's March 2027 PDUFA defines the bull case while nex-z's safety resolution remains open
Gene therapy for a fatal heart disease in FA, with a 13-vs-13 pivotal readout the single gate to a 2028 BLA
Oral brexanolone's second Phase 3 swing at PPD, with BLOOM's site-controlled design the only test of whether the first miss was noise or signal
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
NVG-291 enters registrational RESTORE study in chronic tetraplegia; thesis resolves on whether Phase 2 neural recovery signals replicate at scale
Neumora pivots to NMRA-511 in AD agitation after navacaprant's three Phase 3 failures; the bet now hinges on whether Phase 1b signals translate to Phase 2 efficacy
Scancell-Neuphoria merger bets $79M on iSCIB1+ beating an approved TIL therapy in post-checkpoint melanoma, if Phase 3 confirms the 77% PFS signal
Reverse merger turns Passage Bio into Remix Therapeutics after FDA blocked PBFT02's path; REM-422 Phase 2 ACC data in 2027 is the new make-or-break event
First-ever approved dermatomyositis therapy just launched; commercial uptake now determines whether a multi-indication TYK2/JAK1 platform compounds or stalls
Four years of durable OLE data and Phase 3 enrollment complete; zorevunersen's NDA case hinges on whether Phase 3 confirms the signal
100% responder rate in pivotal trial sets up a single-arm BLA filing; can a novel milestone endpoint survive FDA scrutiny?
FAYUVI becomes Ultragenyx’s second approved gene therapy; Aspire failure still weighs on growth
Filed treatments are listed first. This shows where late-stage treatments are being developed, not which companies will be acquired. Pipeline data and summaries from Readouts as of 29 September 2026; companies already acquired are left out.
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