The biotech sector is on the cusp of a surge in novel therapies. Recent FDA approvals were driven by first-in-class drugs and expedited review paths. Nearly half of 50 new biologic and molecular therapies approved by 2024 will be first-in-class agents. Regulators are accelerating innovative treatments. For example, the Hunter syndrome gene therapy RGX-121 earned Orphan, Rare Pediatric Diseases, Fast Track, and RMAT designations. The AML therapy ziftomenib also received Priority Review and Breakthrough Therapy status, as well as Orphan and Fast Track. The FDA is focusing on areas of high need by increasing the use of Breakthrough and Priority designations. Fast-track pathways combined with novel targets suggest that the FDA will continue to approve pioneering drugs until late 2025.
Here are ten candidates with high impact that should be FDA approved by Q4 of 2025. Each entry includes the drug name, the developer, the indication, any clinical results or status, regulatory designations, and a PDUFA/targeted action date.
- Paltusotine (Crinetics Pharmaceuticals) – Acromegaly: Paltusotine acts as an oral somatostatin type 2 receptor (SST2) antagonist for acromegaly (excess of growth hormone) and gigantism. It’s the first daily pill of its class, designed to maintain IGF-1 levels and symptom control. Phase 3 PATHFNDR-1/2 trials met primary endpoints, sustaining normal IGF-1 and controlling symptoms in treated adults. The NDA was accepted in late 2024, with a target action (PDUFA) date of September 25, 2025. It has Fast Track and orphan designations for acromegaly. If approved, Paltusotine (brand name Palsonify) would replace injectable therapies and fill an unmet need in pituitary disease.
- Ziftomenib (Kura Oncology/Kyowa Kirin) – NPM1‑Mutant AML: Ziftomenib (KO-539) is a first-in-class menin inhibitor for relapsed/refractory acute myeloid leukemia (AML) with NPM1 mutations. Phase 1/2 results (KOMET-001) showed promising remission rates (CR/CRi) in this otherwise hard-to-treat subset. The NDA was accepted in mid-2025, with an FDA decision expected by November 30, 2025 (Priority Review). Ziftomenib holds Orphan Drug designation and Breakthrough Therapy status for NPM1-mutant AML. If approved, it would offer a novel targeted option for ~25% of AML patients, marking a breakthrough in precision oncology.
- Clemidsogene Lanparvovec (RGX-121, Regenxbio) – Hunter Syndrome (MPS II): RGX-121 is a one-time gene therapy for severe Hunter syndrome (mucopolysaccharidosis II). It delivers a functional iduronate-2-sulfatase (IDS) gene via an AAV9 vector to the central nervous system and body. Phase 3 trials met primary endpoints (reducing GAG buildup) and showed cognitive benefits in pediatric patients. The BLA is under Priority Review, with a PDUFA date of November 9, 2025. It has multiple special designations – Orphan, Rare Pediatric, Fast Track, and RMAT. Clemidsogene lanparvovec represents the first systemic gene therapy for Hunter syndrome, potentially transforming a life-threatening pediatric disease.
- Epioxa (Glaukos Corporation) – Keratoconus: Epioxa (formerly ATI-502) is a novel corneal cross-linking solution for keratoconus (a progressive eye disorder causing corneal thinning). Unlike standard procedures, it is an epi-on topical formulation applied in-office to strengthen corneal collagen without removing the epithelium. Phase 3 data showed it halted disease progression. The NDA was accepted, with a PDUFA date of October 20, 2025. While not designated as an orphan, keratoconus is a rare eye condition. Epioxa would be first-in-class as a non-invasive treatment for keratoconus, offering a safer alternative to existing laser surgery approaches.
- Aficamten (Cytokinetics) – Obstructive Hypertrophic Cardiomyopathy (oHCM): Aficamten (formerly CK-274) is an oral cardiac myosin inhibitor for symptomatic obstructive HCM. In Phase 3 (SEQUOIA-HCM), aficamten improved exercise capacity and heart function, reducing outflow obstruction. Cytokinetics filed its NDA, and the FDA extended the PDUFA to December 26, 2025 (to allow a REMS safety review). It has Fast Track and Orphan designations (HCM is a rare disease). Aficamten (brand Olipudase) follows mavacamten (Camzyos) with a similar mechanism; an approval would expand oral therapy options for HCM patients, a high-need cardio condition.
- Sibeprenlimab (Otsuka/Visterra) – IgA Nephropathy: Sibeprenlimab (VIS649) is a first-in-class monoclonal antibody targeting APRIL, a cytokine involved in IgA production. It’s designed to reduce pathogenic IgA deposition in kidney tissue. Phase 3 (VISIONARY) met its interim endpoint: significant reduction in proteinuria vs placebo. The BLA is under Priority Review, with a PDUFA date of November 28, 2025. It has Breakthrough Therapy and Fast Track status for IgA nephropathy. Approval would give patients the first targeted therapy for this chronic kidney disease, enabling convenient monthly subcutaneous dosing.
- Icotrokinra (JNJ‑2113, Janssen) – Plaque Psoriasis: Icotrokinra is an oral small molecule IL-23 receptor inhibitor for moderate-to-severe plaque psoriasis. It is the first oral agent targeting IL-23. Phase 3 ICONIC trials showed robust PASI90 skin clearance, rivalling injectable biologics. Janssen submitted the NDA in July 2025; FDA review (likely Priority Review) should wrap up late 2025 or early 2026. Icotrokinra received Breakthrough Therapy designation. If approved, it would be a first-in-class oral biologic for psoriasis, expanding options beyond injectable monoclonals.
- Orforglipron (LY3502970, Lilly) – Obesity: Orforglipron is an investigational oral GLP-1 receptor agonist for chronic weight management. In a Phase 2 trial, it delivered ~10–15% mean bodyweight loss over 26 weeks, comparable to injectable GLP-1s. Lilly plans to submit an NDA by late 2025. Though not FDA-designated yet (it’s a new endocrine therapy), it represents next-gen weight-loss therapy. Orforglipron’s oral form could dramatically expand GLP-1 access (no injections) if approved. A decision may come in 2026, but it’s worth watching given its pipeline prominence.
- Reproxalap (Aldeyra Therapeutics) – Dry Eye Disease: Reproxalap is an innovative small molecule called “RASP modulator” (reactive Aldehyde Species), for treating dry eye disease (ocular Surface Inflammation). This is the first of its kind and targets inflammation pathways. Aldeyra submitted the NDA again after a pivotal success study in treating severe dry eye symptoms. FDA has set a PDUFA deadline of 16 December 2025. If approved, the topical eyedrops (reproxalap) would be a new treatment for dry eyes in many years. This product will address a huge unmet market.
- Relacorilant (Corcept Therapeutics) – Cushing‘s Syndrome: Relacorilant is a selective oral cortisol moderator for endogenous Cushing syndrome (hypercortisolism). It is a selective glucocorticoid antagonist. The Phase 3 GRACE study met its key endpoints, including significant improvements in blood glucose and blood pressure control when compared to placebo. The NDA has been accepted with Orphan Drug Status and a PDUFA Target Date of December 30, 2020. Relacorilant, also known as Korlym, would be a life-changing treatment for Cushing’s disease patients. Many of them have no other long-term options beyond surgery.
These candidates have all shown impressive trial results, and FDA expedited reviews for each of them. Clinical development professionals and those in regulatory affairs need to be aware of the dates on their PDUFAs and the results of their trials. Approvals of novel therapies (oral IL23, gene therapy and GLP-1 agonists) will reshape treatment paradigms in oncology, rare diseases, endocrinology, and immunology. The treatment paradigms in oncology will be reshaped across rare diseases, immunology, endocrinology and endocrinology.
Next-Generation Weight Loss Drugs: GLP-1, Dual-Agonists and Beyond
After a renaissance led by semaglutide (Ozempic/Wegovy) and tirzepatide (Mounjaro/Zepbound), the next wave of obesity drugs is already emerging. New GLP-1 receptor agonists and combination agonists are in late-stage development, targeting even greater efficacy. For example, Lilly’s retatrutide is a triple-receptor agonist (GLP-1, GIP and glucagon) that in Phase 2 drove up to ~17.5% mean weight loss at 24 weeks (24.2% at 48 weeks) in people with obesity. Another is orforglipron, Lilly’s oral GLP-1 analog, which achieved about 10–15% weight loss in trials. These mechanisms work by suppressing appetite, slowing gastric emptying and boosting metabolism, addressing hunger at its hormonal roots. Beyond Lilly, other candidates include Roche/Pfizer’s efpeglenatide (GLP-1 agonist) and dual-agonists combining GLP-1 with GIP or glucagon. The market potential is immense: analysts project GLP-1 drugs will generate ~50 billion USD in sales by 2025. For biotech stakeholders, tracking this pipeline is critical, as novel weight-loss “super-agonists” may gain FDA approval by late 2025 or 2026 and fundamentally expand the obesity therapy market.
GLP-1 Market Overview: Size, Players, and Growth
The global market for GLP-1 receptor agonists is exploding. By some estimates, the four leading GLP-1 therapies (two each from Novo Nordisk and Lilly) will total about $50 billion in sales by 2025. More broadly, the GLP-1 agonist market was ~$52 billion in 2024 and is projected to grow from ~$62.8 billion in 2025 to roughly $156.7 billion by 2030. Key drivers include the obesity/diabetes epidemic and expanding uses (e.g., NASH, heart failure). Major players dominate this market: Novo Nordisk (Ozempic, Wegovy), Eli Lilly (Mounjaro, Zepbound) and Sanofi, AstraZeneca and others are all investing heavily in GLP-1 R&D. The chart above (by Grand View Research) illustrates regional growth projections – North America leads usage, with Asia-Pacific markets surging as obesity rises. Investors are keen: venture funds and pharma are pouring money into GLP-1 and related metabolic programs. With such rapid growth, clinicians and industry execs are tracking GLP-1 developments closely, from pipeline molecules to new indications.
Conclusion
It is important for biotech professionals to stay abreast of market trends and FDA approvals. In late 2025, the FDA will approve many best-in-class and first-in-class therapies in diverse fields. It is vital that those involved in clinical development and regulatory affairs, as well as commercialization, keep track of these new drugs, from weight-loss agonists to gene therapies. These approvals not only affect patient care, but also investment and competitive strategies. GLP-1 is a good example of how approvals for a few products can lead to a multi-billion-dollar market almost overnight. Understanding upcoming FDA decisions and fast-track designations allows experts to anticipate healthcare shifts. Recent case studies have shown that a single novel approval could redefine a therapeutic area. This is why professionals place a high priority on pipeline intelligence and FDA timelines when planning their strategy.
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