Accelerating Access and Innovation in Global Healthcare
Expedited drug approval pathways—such as FDA Fast Track, Accelerated Approval, and Priority Review—speed up access to treatments for serious diseases, often cutting approval timelines by months or even years. However, these pathways may rely on limited clinical evidence, including surrogate markers rather than direct patient outcomes, which can create uncertainty about long-term safety and effectiveness.
Major Effects of Expedited Drug Approvals:
- Quicker Patient Access: Patients with urgent unmet medical needs can receive new therapies sooner.
- Smaller Evidence Base: Approvals may rely on early-stage trial data rather than full Phase 3 studies.
- Use of Surrogate Measures: Decisions are often based on biomarkers or lab results that may not fully reflect real clinical benefit.
- Potential Safety Risks: Some side effects or efficacy concerns may only emerge after wider public use.
- More Post-Approval Studies: Drug makers are typically required to complete confirmatory trials after approval.
- Higher Treatment Costs: These drugs may enter the market at premium prices despite limited long-term evidence.
- Greater Chance of Withdrawal: If later studies fail to confirm benefit, approvals may be revoked.
2026 Landscape of Expedited Approvals & Designations
In early 2026, expedited drug approvals and regulatory designations are increasingly centered on innovative biologics, advanced therapies, and rare disease treatments. Agencies such as the FDA are streamlining approval pathways to bring targeted therapies to patients more quickly, with accelerated approvals becoming more common—especially in rare diseases and oncology, which continue to dominate breakthrough designations.
Key Regulatory Trends in 2026:
- Greater Focus on Rare Diseases: Rare disease therapies are receiving expanded regulatory support, with many gaining orphan drug designation.
- AI-Powered Drug Development: Artificial intelligence is speeding up drug discovery and helping identify promising candidates for breakthrough designation faster.
- Growth in Precision Immunology: Research is increasingly targeting monoclonal antibodies and fusion proteins aimed at immunology and autoimmune conditions, including BAFF-R, OX40, and IL-36R pathways.
- Expansion of Advanced Therapies: Gene editing, mRNA technologies, and allogeneic cell therapies are growing rapidly, with an emphasis on curative, one-time treatments.
- Stronger Post-Approval Oversight: As accelerated approvals rely more on surrogate endpoints, regulators are tightening post-marketing trial requirements to verify long-term clinical benefit.
Regulatory Approvals/Designations in the first half of 2026
Fast Track Designations
As of May 2026, the total number of Fast Track designations for the year has not yet been finalised or officially published, since the year is still ongoing. However, some notable drugs that have received Fast Track designation include:
1. Enzalutamide Implant (Enolen)- On January 8, 2026, the U.S. FDA granted Fast Track designation to Enolen for the treatment of patients with low- to intermediate-risk localized prostate cancer.
2. Opamtistomig (REOLYSIN)- On January 14, 2026, the U.S. FDA granted Fast Track designation to opamtistomig (LBL-024) for the treatment of extrapulmonary neuroendocrine carcinoma (EP-NEC).
3. BNT113- On January 21, 2026, the U.S. FDA granted Fast Track designation for its mRNA cancer immunotherapy candidate BNT113, for the treatment of patients with PD-L1–expressing, HPV16-positive head and neck squamous cell carcinoma (HNSCC).
4. IBI3003 - On January 26, 2026, the U.S. FDA granted Fast Track designation to IBI3003 for the treatment of relapsed or refractory multiple myeloma (RRMM).
5. LBL-034- On January 28–29, 2026, the U.S. FDA granted Fast Track designation to LBL-034, an investigational GPRC5D/CD3 bispecific T-cell engager, for the treatment of relapsed or refractory multiple myeloma.
6. Arsenic trioxide oral (QTX-2101)- On January 29, 2026, the U.S. FDA granted Fast Track designation to QTX-2101 for the treatment of acute promyelocytic leukaemia (APL).
7. ETX-19477- On January 8, 2026, the U.S. FDA granted Fast Track designation to ETX-19477, a PARG inhibitor, for the treatment of BRCA-mutated, platinum-resistant, high-grade serous ovarian cancer.
8. CTx001- On January 8, 2026, the U.S. FDA granted Fast Track designation to its lead gene therapy candidate, CTx001, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
9. STX-0712- On May 27, 2026, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to STX-0712, an investigational therapy being developed for the treatment of relapsed or refractory chronic myelomonocytic leukemia (CMML). CMML is an aggressive hematologic malignancy with limited therapeutic options, particularly for patients whose disease has relapsed or failed to respond to existing treatments.
10. DOC1021- On May 6, 2026, the U.S. FDA granted Fast Track designation to DOC1021, an investigational patient-derived dendritic cell immunotherapy developed by Diakonos Oncology for the treatment of unresectable or metastatic cutaneous melanoma. This marks the third Fast Track designation awarded to DOC1021 across multiple oncology indications, underscoring the ongoing unmet need in advanced solid tumors.
11. Pelareorep (Reolysin)- On February 4, the U.S. FDA granted Fast Track designation to pelareorep, an immunotherapy, for use in combination with bevacizumab (Avastin) and the FOLFIRI regimen (leucovorin, fluorouracil, and irinotecan) to treat patients with KRAS-mutated, microsatellite-stable metastatic colorectal cancer in the second-line setting.
12. Irpagratinib- On February 10, the U.S. FDA granted Fast Track designation to irpagratinib, a highly selective small-molecule FGFR4 inhibitor, for the treatment of patients with hepatocellular carcinoma (HCC) exhibiting FGF19 overexpression who have previously received immune checkpoint inhibitor and multitargeted kinase inhibitor therapies.
13. PLT012- On February 19, the U.S. FDA granted Fast Track designation to PLT012, an anti-CD36 monoclonal antibody, for the treatment of patients with hepatocellular carcinoma (HCC). The therapy is also being investigated in other solid tumors where existing immunotherapies may offer limited benefit.
14. ART6043- On February 23, the U.S. FDA granted Fast Track designation to ART6043, a DNA polymerase theta inhibitor, in combination with olaparib (Lynparza), a PARP inhibitor, for the treatment of adult patients with BRCA-mutated, HER2-negative locally advanced or metastatic breast cancer who have not previously received PARP inhibitor therapy.
15. AKY-1189- On February 24, the U.S. FDA granted Fast Track designation to AKY-1189, a nectin-4–targeted miniprotein radioconjugate, for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (UC) who have progressed on or after prior systemic therapies.
16. SRN-101- Also on February 24, the U.S. FDA granted Fast Track designation to SRN-101, an immunogene therapy, for the treatment of patients with high-grade glioma.
17. Elunetirom- On May 27, 2026, the US FDA granted Fast Track designation to elunetirom as an adjunctive treatment for depressive episodes in adults with bipolar I or bipolar II disorder.
18. Suplexa- On May 15, 2026, the U.S. FDA granted Fast Track designation to Suplexa for the treatment of patients with microsatellite instability-high (MSI-H) colorectal cancer.
19. Zocilurtatug pelitecan- On May 12, 2026, the U.S. FDA granted Fast Track designation to zocilurtatug pelitecan (zoci; formerly ZL-1310) for the treatment of patients with extrapulmonary neuroendocrine carcinomas (epNECs) whose disease has progressed following standard first-line therapy.
Breakthrough Therapy Designations
Based on early 2026 data, several drugs received Breakthrough Therapy Designation (BTD) through May 2026. The total number of BTDs for the full year has not yet been finalized or officially published, as 2026 is still ongoing.
However, based on publicly disclosed announcements and press releases, some of the most notable Breakthrough Therapy designations include:
1. Pemvidutide - On January 5, 2026, the US FDA granted Breakthrough Therapy Designation to pemvidutide for the treatment of metabolic dysfunction–associated steatohepatitis (MASH).
2. Sevabertinib (Hyrnuo)- On January 6, 2026, both the U.S. FDA and China’s Center for Drug Evaluation (CDE) granted Breakthrough Therapy Designation (BTD) to sevabertinib as a first-line treatment for patients with HER2-mutant non-small cell lung cancer (NSCLC).
3. Privosegtor- On January 6, 2026, the U.S. FDA granted Breakthrough Therapy Designation to privosegtor (formerly OCS-01) for the treatment of acute optic neuritis.
4. IPN60340- On January 13, 2026, the U.S. FDA granted Breakthrough Therapy Designation (BTD) to IPN60340 (also known as ICT01).
5. Zoldonrasib (RMC-9805)- On January 8, 2026, the U.S. FDA granted Breakthrough Therapy Designation to zoldonrasib (RMC-9805) for the treatment of patients with KRAS G12D–mutated non-small cell lung cancer (NSCLC).
6. Alixorexton (ALKS-2680)- On January 6, 2026, the U.S. FDA granted Breakthrough Therapy Designation to alixorexton for the treatment of narcolepsy type 1 (NT1).
7. Emiltatug ledadotin- On May 12, 2026, the US FDA granted Breakthrough Therapy Designation to emiltatug ledadotin (emi-le) for the treatment of patients with locally advanced, recurrent, or metastatic adenoid cystic carcinoma (ACC) exhibiting solid histology or high-grade transformation.
8. Picibanil biosimilar (TARA-002)- On January 5, 2026, the U.S. FDA granted Breakthrough Therapy Designation to TARA-002, an investigational cell-based therapy, for the treatment of pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs).
9. Litifilimab (BIIB-059)- On January 28, 2026, the U.S. FDA granted Breakthrough Therapy Designation to Biogen’s litifilimab for the treatment of cutaneous lupus erythematosus (CLE).
10. Sofetabart mipitecan (MBK-103) - On January 20, 2026, sofetabart mipitecan (LY4170156) received Breakthrough Therapy designation from the U.S. FDA for the treatment of adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have previously been treated with bevacizumab and mirvetuximab soravtansine.
11. Amivantamab and hyaluronidase-lpuj injection (RYBREVANT FASPRO)- On February 18, 2026, the U.S. FDA granted Breakthrough Therapy designation to subcutaneous amivantamab and hyaluronidase-lpuj (RYBREVANT FASPRO™) for the treatment of advanced head and neck squamous cell carcinoma (HNSCC). The designation applies to patients with HPV-unrelated, recurrent or metastatic HNSCC who have progressed following platinum-based chemotherapy and PD-1/PD-L1 inhibitor therapy.
12. Sevabertinib (Hyrnuo)- On January 6, the U.S. FDA granted Breakthrough Therapy designation to sevabertinib (Hyrnuo), a tyrosine kinase inhibitor, for the first-line treatment of patients with locally advanced or metastatic HER2-mutant non-small cell lung cancer (NSCLC).
13. Calderasib (MK-1084)- On May 29, 2026, the US FDA granted Breakthrough Therapy Designation to calderasib (MK-1084), an investigational KRAS G12C inhibitor, for the treatment of certain patients with newly diagnosed metastatic KRAS G12C-mutated non-small cell lung cancer (NSCLC).
Accelerated Approvals
As of early 2026, the total number of U.S. FDA Accelerated Approvals has not yet been formally consolidated, as reporting typically occurs every quarter. However, initial trends indicate a continued emphasis on rare diseases, pediatric conditions, and advanced therapies such as enzyme replacement and gene therapies.
Notable accelerated approvals in the January–May 2026 period include:
1. Tividenofusp alfa (Avlayah)– approved in March 2026 for mucopolysaccharidosis type II (Hunter syndrome), representing progress in treating rare genetic disorders.
2. Navepegritide (Yuviwel)– approved in February 2026 for children with achondroplasia, highlighting innovation in growth disorders.
3. Pegzilarginase (Loargys)– approved in February 2026 for arginase 1 deficiency, a rare metabolic condition.
4. Marnetegragene autotemcel (Kresladi)– approved in early 2026 for a rare pediatric genetic disorder, reflecting the growing role of gene therapies in accelerated pathways.
5. Linerixibat (Lynavoy)- approved on March 17, 2026, for the treatment of cholestatic pruritus in adults with primary biliary cholangitis (PBC).
6. Icotrokinra (Icotyde)- approved on March 17, 2026, for the treatment of moderate-to-severe plaque psoriasis in adults and pediatric patients (12 years of age and older) who weigh at least 40 kg and are candidates for systemic therapy or phototherapy.
7. Bulevirtide (Hepcludex)- approved on May 22, 2026, the First and Only Approved Treatment for Chronic Hepatitis Delta Virus (HDV).
8. Pivekimab sunirine (Decnupaz)- approved on May 27, 2026. approved for the treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN).
9. Sonrotoclax- On May 13, 2026, the U.S. Food and Drug Administration granted accelerated approval to sonrotoclax (Beqalzi, BeOne Medicines USA, Inc.), a BCL-2 inhibitor, for the treatment of adults with relapsed or refractory mantle cell lymphoma (MCL) following at least two prior lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor.
Overall, early 2026 data suggest that accelerated approvals remain highly concentrated in orphan and speciality indications, with a strong pipeline of biologics and next-generation therapies aimed at addressing unmet medical needs.
Priority Review
Priority Review designations continued to play a pivotal role in accelerating the development and regulatory assessment of therapies addressing significant unmet medical needs in 2026. Regulatory agencies worldwide, including the U.S. FDA, Health Canada, and China's NMPA, granted Priority Review status to several promising treatments across diverse therapeutic areas such as oncology, rare diseases, immunology, infectious diseases, endocrinology, and metabolic disorders. Notable recipients included linerixibat for cholestatic pruritus, bepirovirsen for hepatitis B, dupilumab for allergic fungal rhinosinusitis, neladalkib for ALK-positive NSCLC, and multiple innovative therapies targeting chronic kidney disease, generalised myasthenia gravis, severe hypertriglyceridemia, thyroid eye disease, and gastrointestinal stromal tumors. These designations underscore the ongoing commitment to expediting patient access to potentially transformative medicines.
Key Priority Review in 2026:
1. Linerixibat (Lynavoy)- On March 17, 2026, linerixibat received priority review for the treatment of cholestatic pruritus in patients with primary biliary cholangitis in China.
2. Bepirovirsen- On Apr 30, 2026, Health Canada granted priority review status for the treatment of hepatitis B.
3. Venglustat- Granted Priority Review for the treatment of thyroid eye disease (TED), with a target action date of June 30, 2026.
4. Finerenone (Kerendia)- In May 2026, the U.S. FDA accepted and granted priority review to the supplemental NDA for KERENDIA (finerenone) for the treatment of adults with type 1 diabetes-associated chronic kidney disease (CKD).
5. Dupilumab- On February 28, 2026, the US FDA granted Priority Review to the supplemental Biologics License Application (sBLA) for dupilumab for the treatment of allergic fungal rhinosinusitis (AFRS), a subtype of chronic rhinosinusitis, in adults and children aged 6 years and older. AFRS is a chronic type 2 inflammatory sinus disease driven by an exaggerated immune response to fungal allergens.
6. Neladalkib- On May 27, 2026, the US FDA accepted the New Drug Application (NDA) for neladalkib and granted it Priority Review for the treatment of patients with advanced ALK-positive non-small cell lung cancer (NSCLC) who have previously received tyrosine kinase inhibitor (TKI) therapy.
7. Pembrolizumab Combinations- On April 20, 2026, the US FDA granted Priority Review to KEYTRUDA® (pembrolizumab) and KEYTRUDA QLEX™ (pembrolizumab and berahyaluronidase alfa-pmph), each in combination with Padcev® (enfortumab vedotin-ejfv), for the treatment of cisplatin-eligible patients with muscle-invasive bladder cancer.
8. Efgartigimod alfa-fcab- In January 2026, the US FDA accepted and granted Priority Review to a supplemental Biologics License Application (sBLA) for VYVGART® for the treatment of adults with acetylcholine receptor antibody (AChR-Ab) seronegative generalized myasthenia gravis (gMG).
9. Olezarsen- In February 2026, the US Food and Drug Administration (FDA) accepted for Priority Review the supplemental New Drug Application (sNDA) for olezarsen for severe hypertriglyceridemia (sHTG).
10. Finerenone (KERENDIA)- In May 2026, the U.S. FDA accepted and granted priority review to the supplemental NDA for KERENDIA (finerenone) for the treatment of adults with type 1 diabetes-associated chronic kidney disease (CKD).
11. Bezuclastinib/Sunitinib- On May 29, 2026, the US FDA accepted and granted Priority Review to a New Drug Application (NDA) for bezuclastinib (CGT9486) in combination with sunitinib (Sutent) for the treatment of patients with gastrointestinal stromal tumors (GIST) who have previously been treated with imatinib (Gleevec).
12. Saroglitazar- On May 28, 2026, the US FDA granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 27, 2026, for the application seeking approval for the treatment of Primary Biliary Cholangitis (PBC).
Orphan drug Designations
Several therapies were granted Orphan Drug Designation (ODD) in early 2026 across areas such as oncology, immunology, and metabolic disorders, reflecting a continued focus on rare conditions with significant unmet needs. Notable examples include Pegrizeprument (VEL-101) for preventing transplant rejection, Rilzabrutinib for IgG4-related disease, and Zenocutuzumab-zbco for rare bile duct cancer.
Key Orphan Drug Designations in 2026:
1. Pegrizeprument (VEL-101)- On January 21, 2026, the U.S. Food and Drug Administration granted Orphan Drug Designation to pegrizeprument (VEL-101) for the prevention of organ rejection in liver transplant recipients.
2. Rilzabrutinib- Rilzabrutinib has been granted orphan drug designation for IgG4-related disease (IgG4-RD) across multiple regions, including the United States (April 1, 2025), the European Union (August 14, 2025), and Japan, where the Ministry of Health, Labour and Welfare approved the designation on March 2, 2026. The therapy is being developed to address this rare condition, which has significant unmet medical needs.
3. Zenocutuzumab-zbco (BIZENGRI)- On February 5, 2026, the U.S. Food and Drug Administration granted Orphan Drug Designation (ODD) to BIZENGRI (zenocutuzumab-zbco) for the treatment of advanced, unresectable, or metastatic cholangiocarcinoma with NRG1 gene fusions, specifically in patients whose disease has progressed following prior systemic therapy.
4. Dusquetide (SGX945)- Dusquetide (SGX945) was granted orphan drug designation by the European Commission on March 26, 2026, for the treatment of Behçet’s disease. Earlier, the U.S. Food and Drug Administration had awarded orphan status on August 13, 2025, following positive Phase 2a results.
5. Morcamilast (ME3183)- Meiji Seika Pharma's morcamilast (ME3183) was granted orphan medicinal product designation by the European Commission (EC) for the treatment of palmoplantar pustulosis (PPP) on March 3, 2026.
6. (Z)-Endoxifen- (Z)-Endoxifen was granted Orphan Drug Designation by the U.S. Food and Drug Administration on January 15, 2026, for the treatment of Duchenne muscular dystrophy (DMD), with the announcement made public on January 16, 2026. The therapy had previously received orphan designation for DMD in the European Union on July 18, 2025.
7. CK0804- On January 6, the U.S. FDA granted Orphan Drug designation to CK0804, an investigational allogeneic, off-the-shelf regulatory T-cell (Treg) therapy, for the treatment of myelofibrosis. The designation was supported by findings from a study of 13 heavily pretreated patients, in which CK0804 demonstrated reductions in spleen volume and symptom burden, along with improvements in transfusion requirements.
8. LP-284- Lantern Pharma’s LP-284 was granted Orphan Drug Designation by the U.S. Food and Drug Administration on January 20, 2026, for the treatment of soft tissue sarcomas. The designation supports the development of this small-molecule, synthetic lethal therapy for rare cancers.
9. NP-G2-044- Novita Pharmaceuticals announced that the U.S. Food and Drug Administration granted Orphan Drug Designation to its oral fascin inhibitor, NP-G2-044, on January 12, 2026, for the treatment of pancreatic cancer. The designation covers its use both as a monotherapy and in combination with anti-PD-1 immune checkpoint inhibitors for this indication.
10. CPI-008- On January 7, the U.S. FDA granted Orphan Drug designation to CPI-008, an imaging agent used for intraoperative margin detection in pancreatic cancer. CPI-008 has demonstrated strong imaging performance in investigator-initiated Phase 2 studies across several cancers, including pancreatic, head and neck, and colorectal cancers.
11. ARB1002- On January 7, the US FDA granted Orphan Drug designation to ARB1002, an investigational antibody-drug conjugate (ADC) targeting CDH17, for the treatment of pancreatic cancer.
12. Linerixibat (Lynavoy)- In March 2026, linerixibat was granted Orphan Drug Designation for the treatment of cholestatic pruritus in patients with primary biliary cholangitis in Japan.