Which small-molecule drugs are making the mark in 2026 in new drug approvals or in recently or soon-to-be launched drugs? DCAT Value Chain Insights takes an inside look.

By Patricia Van Arnum, Editorial Director, DCAT, [email protected]

Small-molecule drugs making the news
How are small-molecules faring in 2026? To answer that question, DCAT Value Chain Insights examined where the small-molecules drugs among the 11 drugs identified by Clarivate, a business intelligence firm, in its 2026 Drugs To Watch report. The annual analysis, released in January (January 2026), identified 11 drugs to watch in 2026–recently launched or likely to enter the market or meet a consequential milestone in 2026 and forecast to become blockbusters (defined as reaching sales of $1 billion or more) within five years (by 2031) and/or to transform treatment paradigms.

Of these 11 drugs, seven were small molecules, and four of those small-molecule drugs have been approved by the US Food and Drug Administration (FDA)—Eli Lilly and Company’s Foundayo (orforglipron), Celcuity’s Revtorpy (gedatolisib), Corcept Therapeutics’ Lifyorli (relacorilant), and Johnson & Johnson’s Inlexzo (gemcitabine intravesical system). The three other small-molecule drugs from that list of drugs to watch—Sanofi’s (tolebrutinib), Bristol-Myers Squibb’s (BMS’) mezigdomide, and BeOne Medicines’s BGB-16673 (tacabrutideg)—have not yet been approved by FDA. Where do these seven small-molecule drugs now stand, and what is differentiating them in the market?

Driver in the obesity market
On top of the list is Eli Lilly and Company’s Foundayo (orforglipron), an oral obesity drug, which was approved by FDA in April (April 2026) and is in late-stage development for treating Type 2 diabetes. Foundayo is a once-daily small-molecule (non-peptide) oral glucagon-like peptide-1 (GLP-1) receptor agonist (RA). Analysts project blockbuster potential for the drug with the oral administration being a key driver for revenue potential. In a recent analysis, Clarivate projects sales of orforglipron of $11.1 billion for obesity in 2031 in the G7 markets (Canada, France, Germany, Italy, Japan, the UK, and the US) and $5.2 billion in expected sales for Type 2 diabetes in the G7 markets in 2031. Orforglipron was the first oral, non-peptide, small-molecule GLP-1 RA to be approved by FDA and be taken without food or water restrictions at any time of the day. As such, it represents a breakthrough in convenience and accessibility compared with injectable alternatives, according to the Clarivate analysis.

Rethinking the paradigm in women’s health
Three of the small-molecule drugs in Clarivate’s 2026 Drugs to Watch and approved by FDA represent advances in women’s health, in defining women’s health as not only female-specific conditions but also health conditions disproportionately affecting women or affecting women differently than men. These include Celcuity’s Revtorpy (gedatolisib), Corcept Therapeutics’ Lifyorli (relacorilant), and J&J’s Inlexzo (gemcitabine intravesical system).

The key differentiator of J&J’s Inlexzo (gemcitabine intravesical system) is the delivery system, an intravesical drug-releasing system that provides extended local delivery of a cancer medication into the bladder as a bladder-sparing option performed in an outpatient setting. The device then remains in the bladder for about three weeks to release gemcitabine continuously. It was approved in the US in 2025 for the treatment of adult patients with Bacillus Calmette-Guérin unresponsive, non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors. It is also being evaluated to treat muscle-invasive bladder cancer. Clarivate projects sales of $1.8 billion in the G7 markets in 2031. 

Celcuity’s Revtorpy (gedatolisib) is an intravenous drug for treating hormone receptor (HR)-positive/ human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer. The drug is a kinase inhibitor indicated in combination with fulvestrant, with or without palbociclib, for HR-positive, HER2-negative locally advanced or metastatic breast cancer in adult patients whose tumors do not have a PIK3CA mutation and whose cancer has progressed after endocrine (hormone) therapy. It was approved by FDA in July (July 2026). Outside of women’s health, it is also being evaluated for metastatic castration-resistant prostate cancer. Clarivate projects sales of $1.08 billion in the G7 markets in 2031.

Corcept Therapeutics’ Lifyorli (relacorilant) is a selective cortisol modulator for treating patients with platinum-resistant ovarian cancer and was approved by FDA in March (March 2026).  Platinum-resistant ovarian cancer has limited, often short-lived, treatment options, with current therapies offering only modest benefit, and many are restricted by biomarker requirements, according to the Clarivate analysis. Relacorilant addresses this gap by targeting cortisol-driven chemoresistance. Clarivate projects sales of $150 million by 2031 and is noted for its scientific advances in treatment. The drug is also being evaluated to treat endogenous hypercortisolism (Cushing syndrome) and hypercortisolism associated with adrenal adenoma or hyperplasia and  prostate cancer.

Oncology and protein degraders
One of the ongoing areas of drug development is to tackle so-called “undruggable” drugs, which refers to certain disease-associated proteins that lack traditional binding pockets, thereby making them highly difficult to target with conventional small-molecule drugs. Targeted protein degradation, which uses the cell’s innate protein degradation mechanisms to selectively down-regulate or eliminate pathogenic proteins, is a promising area to address that problem. Clarivate points to two protein degraders in oncology: BMS’ mezigdomide and BeOne Medicines’s BGB-16673 (tacabrutideg).

BMS’ mezigdomide is an oral drug to treat relapsed or refractory multiple myeloma. It is part of the company’s lifecycle management strategy against generics incursion to replace the company’s Imnovid/Pomalyst (pomalidomide). Its other investigational multiple myeloma treatment, iberdomide, is being positioned as a more tolerable replacement for the company’s Revlimid (lenalidomide).In July (July 2026), FDA  accepted a new drug application (NDA) for mezigdomide in combination with carfilzomib and dexamethasone in patients with relapsed or refractory multiple myeloma. FDA has granted a Prescription Drug User Fee Act (PDUFA) date of May 13, 2027, for this indication. Clarivate projects expected sales of $1.47 billion in the G7 markets in 2031. 

BeOne Medicines’s BGB-16673 (tacabrutideg) has once-daily oral administration to treat relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma. It is also being evaluated for relapsed or refractory Waldenstrom’s macroglobulinemia, a rare B-cell lymphoma, and other relapsed or refractory B-cell malignancies, including mantle cell lymphoma and follicular lymphoma, and chronic spontaneous urticaria. Clarivate projects sales of $1.19 billion in the G7 markets in 2031.

MS drug tolebrutinib
Sanofi’s tolebrutinib is a Bruton tyrosine kinase (BTK) inhibitor and once-daily oral administration to treat non-relapsing secondary progressive-multiple sclerosis. It has a novel mechanism of action that enables crossing the brain–blood barrier and addresses the core inflammatory processes underlying progressive MS, giving it strong potential over other treatments for progressive MS, particularly with the convenience of its oral administration over injectable therapies, according to the Clarivate analysis. The company had a setback for tolebrutinib in late December 2025 with the issuance of a Complete Response Letter by FDA for the NDA for tolebrutinib, when FDA, issued the CRL due to the risk of drug-induced liver injury. At the time, Sanofi said it would work with FDA to address those concerns. Since then, in June (June 2026), the European Commission approved Cenrifki (tolebrutinib) for the treatment of secondary progressive multiple sclerosis without relapses in the last two years.

Other small-molecule drugs making the mark
What other small-molecule drug approvals by FDA in 2026 are of particular market note? Below are drugs targeted by some analysts as blockbuster contenders into the early 2030s.

AstraZeneca’s Baxfendy (baxdrostat), approved by FDA in May (May 2026), is an oral, small-molecule aldosterone synthase inhibitor (ASI) to help lower high blood pressure in adults. Baxfendy is an ASI designed to lower blood pressure in a new way by specifically inhibiting the production of aldosterone, a hormone that raises blood pressure to unhealthy levels and increases the risk of heart and kidney problems. As part of a broad development program, Baxfendy is also being investigated in clinical trials in other conditions where high aldosterone plays a role in elevating cardiorenal risk, including as a monotherapy for primary aldosteronism, and in combination with dapagliflozin for chronic kidney disease and hypertension and the prevention of heart failure in patients with hypertension. AstraZeneca acquired Baxfendy through its acquisition of CinCor Pharma. in 2023.

AstraZeneca also gained FDA approval last month (September 2026) for Etcamah (camizestrant), a small-molecule drug to treat certain forms of breast cancer. It is an estrogen receptor antagonist and was approved in combination with a CDK4/6 inhibitor (abemaciclib, palbociclib, or ribociclib) for adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer upon detection of estrogen receptor-1 (ESR1) mutation during aromatase inhibitor and CDK4/6 inhibitor therapy, based on an FDA-authorized test. The drug was granted accelerated approval and continued approval for this indication may be contingent upon additional confirmatory trials.

Another small-molecule drug approval in 2026 of note is Pfizer’s/Arvinas’/Rigel Pharmaceuticals’ vepdegestrant, a small-molecule PROteolysis Targeting Chimera (PROTAC) protein degrader. The drug is an oral medication for treating ER+/HER2-advanced breast cancer. In June (June 2026), Rigel Pharmaceuticals entered into an exclusive, global license agreement with Arvinas and Pfizer to develop, manufacture, and commercialize Veppanu (vepdegestrant). Rigel is responsible for the launch and commercialization of Veppanu in the US and will own global rights with the ability to sub-license to potential partners to further develop and commercialize Veppanu outside of the US. Arnivas and Pfizer had earlier formed an agreement in 2021 for the co-development and co-commercialization of vepdegestrant.

Up-and-coming for potential launch in 2027 is ulixacaltamide from Praxis Precision Medicines, a Boston-based bio/pharmaceutical company. FDA accepted the company’s NDA for ulixacaltamide for treating essential tremor in adults and set a target action date under the Prescription Drug User Fee Act (PDUFA) of January 29, 2027. Ulixacaltamid is a small-molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical circuit correlated with tremor activity. Ulixacaltamide has received Breakthrough Therapy Designation from  FDA and is the most advanced program within Praxis’ Cerebrum small-molecule platform.

On the rare-disease front, Takeda received FDA approval in August (August 2026) for Orzeyful (oveporexton) for treating narcolepsy Type 1,a rare neuropsychiatric sleep disorder. Oveporexton, developed in-house by Takeda, is part of the company’s orexin franchise aimed at restoring orexin signaling in a variety of neurological conditions.

Travere Therapeutics, a San Diego, California-based bio/pharmaceutical company, received FDA approval in April (April 2026) for Filspari (sparsentan) for reducing proteinuria (abnormal levels of protein in the urine) in patients with focal segmental glomerulosclerosis, a rare kidney disorder.

Priovant Therapeutics received FDA approval for Lisraya (brepocitinib) in August (August 2026) as the first targeted, once-daily oral treatment for adult dermatomyositis, a rare systemic autoimmune disease causing muscle weakness and skin lesions. Priovant Therapeutics is a subsidiary and a so-called “vant” spin-off company majority-owned by Roivant Sciences. Priovant was established in 2021 as a joint venture between Roivant and Pfizer, in which Pfizer licensed oral and topical brepocitinib’s global development rights and US and Japan commercial rights to Priovant. Pfizer holds an approximate 25% equity ownership interest in Priovant.