(Expedited) Designations for Deals: a Win-Win Strategy?

My previous post listed all the announced deals amid the JP Morgan's Healthcare Conference 2025. But I am more curious about how many the drugs of those deals are associated with expedited designations from Health Authorities like FDA and EMA, and any trends across therapeutic areas.

A little background information, the FDA uses four distinct mechanisms to speed the development and availability of drugs treating serious or life-threatening conditions, which I recapped them below as a quick reference.

·?????? Accelerated Approval allows drugs for serious conditions to be approved based on surrogate endpoints that are reasonably likely to predict clinical benefit.

·?????? Fast track designation is meant to expedite the review of drugs that treat serious conditions and fill an unmet medical need.

·?????? Breakthrough therapy designation is for drugs that show substantial improvement over existing therapies on one or more clinically significant endpoints.

·?????? Priority Review designation asks for potentials and/or evidences of a significant improvement in safety or effectiveness over existing therapies, which in turn will greatly accelerate the NDA review time within six months vs standard ten-month review period.

Orphan drug designation (aka ODD) which is given to drugs intended to treat rare diseases affecting fewer than 200,000 people in the U.S, can offers market exclusivity, tax credits, FDA fee waiver, grant eligibility, and regulatory assistance like more frequent guidance during drug development.

According to my observation, out of the 37 deals and collaborations listed from my previous post, 32 drugs?have been granted or will have great potentials for at least one of expedited program designations or ODD.

I’m grouping expedited designations by therapeutic areas, such as oncology and neurology, to provide a detailed narrative summary. This approach ensures a clear organization and thorough analysis, highlighting key measures for each area. Below is an analysis of the expedited designations grouped by therapeutic areas, along with observations on trends and strategic focuses:

1. Oncology

Oncology represents the largest group among the expedited designations, reflecting the high unmet need and evolving therapeutic innovations in cancer treatment. Key observations include:

• Lung Cancer (NSCLC & SCLC): Teliso?V (AbbVie) and Keytruda (Merck & Co.) have received Accelerated Approvals for lung cancers (including NSCLC), with Keytruda (subcutaneous) expected in 2025. Zidesamtinib & Neladalkib (Nuvalent) and Taletrectinib (Nuvation Bio) are focused on molecularly defined subsets of NSCLC (targeting ROS1/ALK and ROS1, respectively) and have received Breakthrough or Orphan designations. Taletrectinib is already under FDA priority review, with decisions expected by mid?2025. Blenrep (GSK) and B7H3 (GSK) are both designated as Breakthrough therapies targeting small cell lung cancer (SCLC) and, in the case of B7H3, also osteosarcoma.
• Lymphomas and Related Hematologic Malignancies: Epkinly/Tepkinly (Genmab/AbbVie) (for diffuse large B?cell lymphoma [DLBCL] and follicular lymphoma [FL]) and CB?010 (Caribou Biosciences) (for DLBCL) received Accelerated and Fast Track/Orphan designations, respectively. IM?1021 (Immunome), a ROR1?targeted ADC, is under development for both solid tumors and B?cell lymphoma, with a potential pathway to Accelerated Approval. Cemsidomide (C4 Therapeutics) is in early clinical phases (expected Phase II initiation in 2026) for relapsed/refractory peripheral T?cell lymphoma (PTCL).
• Other Solid Tumors: Tivdak (Genmab/Pfizer) received Accelerated Approval for recurrent or metastatic cervical cancer. Ziihera (zanidatamab) (Zymeworks) was granted Accelerated Approval for HER2?positive biliary tract cancer. CDK2i (INCB123667) (Incyte) targets Cyclin E?overexpressing ovarian cancer and has potential for accelerated approval as data are expected in the second half of 2026. R289 (Rigel Pharmaceuticals) and Tebapivat (Agios Pharmaceuticals), targeting lower?risk myelodysplastic syndromes (MDS/LR?MDS), received Fast Track/Orphan and Orphan designations, respectively.
• Rare and Special Tumor Types: Varegacestat (Immunome) received an Orphan designation for desmoid tumors, a rare soft tissue neoplasm.

Summary Observation: There is a pronounced focus on precision therapies (targeting specific molecular alterations such as ROS1, ALK, HER2, and ROR1) and on addressing both common and rare hematologic malignancies. The prevalence of Accelerated Approval and Breakthrough designations underscores the critical need for novel treatments in cancers with limited options.

2. Neurology and Neuromuscular Disorders

This category includes both rare neuromuscular conditions and neurodegenerative disorders:

• Spinal Muscular Atrophy (SMA): OAV101 IT (Novartis) received an Orphan designation, reflecting the continuing effort to address rare motor neuron diseases.
• Duchenne Muscular Dystrophy (DMD): Del?zota (Avidity Biosciences) is set for Accelerated Approval (expected by the end of 2025). Sevasemten (Edgewise Therapeutics), an orally administered skeletal myosin inhibitor, has been granted Fast Track and Orphan Drug designations.
• Other Neurological Conditions: REC?994 (Recursion) was awarded an Orphan designation for cerebral cavernous malformation, a rare neurovascular disorder. ANX005 (Annexon Biosciences) received an Orphan designation for Guillain?Barré syndrome, highlighting a focus on immune?mediated neuropathies. LX2006 (Lexeo Therapeutics) is a gene therapy for Friedreich Ataxia cardiomyopathy. While Friedreich Ataxia is primarily a neurodegenerative disorder, the cardiomyopathy aspect brings a dual focus on neurological and cardiovascular endpoints.

Summary Observation: The designations in this area underscore a dual focus on genetic and rare neuromuscular diseases, with both traditional and gene therapy approaches emerging. Expedited pathways (Orphan, Fast Track, and potential Accelerated Approvals) signal robust innovation aimed at conditions with high unmet needs.

3. Cardiovascular and Metabolic Disorders

A number of expedited programs are targeting cardiovascular conditions, often intertwined with metabolic or systemic disorders:

• Cardiomyopathies: Attruby (acoramidis) (BridgeBio) has received Accelerated Approval for transthyretin amyloid cardiomyopathy (ATTR?CM) based on biomarker improvements. Aficamten (Cytokinetics) is being developed for hypertrophic cardiomyopathy and is under Breakthrough Therapy and Fast Track designations, with potential for Accelerated Approval pending positive trial outcomes.
• Metabolic Liver Disorders: Rezdiffra (resmetirom) (Madrigal Therapeutics) achieved the first Accelerated Approval for non?alcoholic steatohepatitis (NASH), targeting liver fat reduction and fibrosis improvement. LUNAR??OTC (ARCT?810) (Arcturus Therapeutics) is an mRNA therapy for ornithine transcarbamylase (OTC) deficiency, a rare metabolic liver disease, and has been granted multiple designations (Fast Track, Rare Pediatric, Orphan) in both the U.S. and Europe.

Summary Observation: The expedited designations in this segment reflect increasing innovation in both heart diseases (with a focus on cardiomyopathies that often have a genetic or protein misfolding basis) and metabolic liver disorders. The use of biomarkers for accelerated approvals and the emergence of mRNA/gene therapy modalities are notable trends.

4. Hematology

Within hematology, particularly the myelodysplastic syndromes:

• Myelodysplastic Syndromes (MDS): R289 (Rigel Pharmaceuticals) is under Fast Track/Orphan designations for lower?risk MDS. Tebapivat (Agios Pharmaceuticals) was granted an Orphan designation for lower?risk MDS, reinforcing efforts to develop treatments for this patient population.

Summary Observation: The focus on lower?risk MDS indicates an effort to address unmet needs in patient subsets that may not be served by current therapies, with expedited pathways enabling more rapid clinical development.

5. Nephrology

In nephrology, the designations address both kidney function and related complications:

• Chronic Kidney Disease (CKD) and Related Conditions: Vafseo (vadadustat) (Akebia Therapeutics) received an Orphan designation for anemia in CKD. Rilparencel (ProKidney), an autologous cell therapy for chronic kidney disease, is under review for a potential Accelerated Approval based on improvements in estimated glomerular filtration rate (eGFR).

Summary Observation: The therapies under this category emphasize innovative approaches—including cell therapy—to improve renal function and treat complications associated with CKD, highlighting a strategy to move beyond conventional treatments.

6. Other Therapeutic Areas

• Psychiatry: Zurzuvae (Sage Therapeutics) has received Breakthrough Therapy designation for postpartum depression, a critical area in mental health where rapid development is essential.
• Infectious Diseases/Transplant-Related Complications: SER?155 (Seres Therapeutics), a microbiome therapeutic for bloodstream infections in transplant patients, has been granted Breakthrough Therapy designation.
• Rare or Less Common Indications: PRGN?2012 (Precigen), targeting recurrent respiratory papillomatosis (RRP), has received an Orphan designation with a BLA submitted and potential Accelerated Approval anticipated in 2025. Varegacestat (Immunome), while classified under oncology as a treatment for desmoid tumors, also falls into the realm of rare diseases given the limited prevalence of the condition.

Summary Observation: The designations in psychiatry and infectious diseases underscore the FDA’s willingness to fast‐track therapies for conditions that, although may not affect large populations, have significant individual impacts and limited current treatment options. The inclusion of rare and orphan indications further demonstrates the importance of addressing unmet needs in smaller patient populations.

Overall Insights

• Dominance of Oncology: Oncology remains the primary focus for expedited designations, driven by advances in precision medicine and the urgent need for novel treatments in diverse tumor types.
• Innovation in Rare Diseases: Across neurology, hematology, and metabolic disorders, orphan and fast?track designations highlight the push to develop therapies for rare conditions that historically have had few treatment options.
• Integration of Novel Modalities: The range of modalities—from monoclonal antibodies and ADCs to gene and mRNA therapies—indicates that the FDA is supporting a broad spectrum of innovative approaches to address both common and rare diseases.
• Biomarker?Driven Approaches: Several therapies (e.g., Attruby, Rezdiffra) are being developed with biomarker endpoints in mind, facilitating expedited review processes by providing early indicators of clinical benefit.

It's nice to see how the expedited designation strategies are being leveraged to address critical areas of unmet need across a diverse range of therapeutic areas, with oncology and rare diseases leading the way in innovation and regulatory flexibility. My next goal and post will be to dive deeper into those drugs and cross-reference them with deal-makings individually from my previous post, for any insights and trends beyond expedited programs and deal-making logics. Simply put is to whether those expedited program can actually win business deals, where FDA in a way serves as the first layer of lens on the drug before any BD, PE, VC, and bankers jump on the valuation train.