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Where the Next Decade of Non-Oncology Pharmaceutical Growth Is Coming From, and What Could Slow It

Mobility Foresights Healthcare & Life Sciences Research | Hub Analysis | September 2026

Outside oncology, pharmaceutical growth is concentrating in a small number of therapeutic areas where new mechanisms have unlocked very large patient populations. The clearest example is obesity. GLP-1 and dual GIP/GLP-1 agonists have turned a lifestyle condition into a pharmacologically treatable disease, and the SELECT trial in 2023 showed a 20% reduction in major cardiovascular events with semaglutide. The same pattern is repeating on a smaller scale in several other areas:

• Cardiovascular disease, where lipoprotein(a), oral PCSK9 inhibition and cardiac myosin inhibition are extending treatment into new populations.
• Alzheimer's disease, where anti-amyloid antibodies are the first therapies to slow the disease itself.
• Psychiatry, where Cobenfy became the first schizophrenia drug with a new mechanism in decades.
• Rare genetic disease, where CRISPR-based therapies such as Casgevy moved from concept to approved product in 2023.

This hub brings together 97 Mobility Foresights market studies across seven areas: obesity and metabolic disease; cardiovascular therapeutics; neuroscience and CNS; rare and genetic disease; infectious disease and vaccines; plasma-derived therapies; and a group of specialty areas spanning bone, renal, respiratory, urology, women's health and aging.

Key Takeaways

• Obesity is the largest new pharmaceutical market in a generation. The competitive frontier is moving from weekly injectables to oral, monthly, multi-agonist and muscle-preserving therapies.
• Cardiometabolic outcomes data are widening the addressable market. Cardiovascular, kidney and sleep-apnea indications give payers a reason to reimburse beyond weight loss.
• Neuroscience has re-entered a growth phase. Anti-amyloid antibodies, brain-shuttle delivery and novel psychiatric mechanisms are reviving a category many large companies had exited.
• Genetic medicine is shifting from ex vivo to in vivo. Base and prime editing delivered directly into the body could turn one-time cures into scalable products.
• Long-acting formats are the common thread. Twice-yearly HIV prevention, twice-yearly siRNA for cholesterol, long-acting injectable antipsychotics and monthly obesity drugs all compete on dosing convenience.

How We Segment Specialty Pharma Beyond Oncology

1. Obesity, Diabetes and Metabolic Disease

Semaglutide (Wegovy) and tirzepatide (Zepbound) established the category, and demand outran manufacturing capacity for most of 2023 and 2024. Our obesity and metabolic therapeutics market report sizes the total category across obesity, type 2 diabetes and related metabolic conditions. The GLP-1 and incretin therapeutics market study focuses on the incretin class itself, covering GLP-1, GIP and glucagon receptor agonism and the supply and pricing dynamics of the leading brands.

The next generation is defined by five design goals:

• Greater weight loss through multiple receptors. The multi-agonist obesity therapeutics market report covers dual and triple agonists such as retatrutide (GIP, GLP-1 and glucagon).
• Oral dosing. The oral GLP-1 therapeutics market study tracks oral peptides and small molecules such as orforglipron, which reported Phase 3 results in 2025 and could remove injection and cold-chain barriers.
• Less frequent dosing. The ultra-long-acting obesity therapeutics market report covers monthly and longer-interval candidates, including antibody-peptide conjugates.
• Better tolerability through amylin. The amylin-mimicking therapy market study covers amylin analogs, alone and combined with semaglutide, which aim to deliver weight loss with fewer gastrointestinal side effects.
• Muscle preservation. The muscle-preserving obesity therapeutics market report covers activin and myostatin-pathway agents, such as bimagrumab, designed to protect lean mass during rapid weight loss.

Two further reports give the pipeline and derivative view. The next-generation obesity drug market report ranks candidates by mechanism, stage and differentiation. The GLP-1 derivatives and next-gen obesity therapeutics market study focuses on modified peptides and new chemical entities built on the GLP-1 scaffold.

Metabolic liver disease has become the first major spillover indication. Resmetirom (Rezdiffra) became the first approved therapy for MASH in March 2024, and semaglutide followed in 2025. Our non-alcoholic steatohepatitis (NASH/MASH) drug market report covers THR-beta agonists, FGF21 analogs and incretins in liver disease. In diabetes itself, the diabetes combination therapy market study tracks fixed-ratio insulin-GLP-1 products and multi-class oral regimens.

2. Cardiovascular Therapeutics

Cardiovascular disease remains the leading cause of death worldwide, and a new wave of drugs is extending treatment beyond statins. Our atherosclerotic cardiovascular disease (ASCVD) drug market report sizes lipid-lowering and anti-inflammatory therapies for ASCVD. It covers statins, ezetimibe and bempedoic acid, PCSK9 antibodies and the twice-yearly siRNA inclisiran.

Two lipid targets are drawing most of the investment:

• Oral PCSK9 inhibition. PCSK9 antibodies lowered LDL cholesterol dramatically but achieved modest uptake because they are injected. The oral PCSK9 inhibitor market report covers oral macrocyclic peptides and small molecules in Phase 3, which could bring antibody-level LDL lowering to a daily pill.
• Lipoprotein(a). Elevated Lp(a) is a genetically determined risk factor affecting roughly one in five people, with no approved therapy. The lipoprotein(a) therapeutics market study covers the antisense, siRNA and oral candidates now in cardiovascular outcomes trials.

Cardiomyopathy is the second growth pocket. Mavacamten (Camzyos), approved in 2022, validated cardiac myosin inhibition in obstructive hypertrophic cardiomyopathy, and aficamten reported positive Phase 3 results in 2024. Our cardiac myosin inhibitors market report covers the class. The broader next-generation cardiomyopathy drugs market study adds transthyretin amyloid cardiomyopathy, where stabilizers and gene silencers such as acoramidis and vutrisiran have joined tafamidis.

Five reports provide the portfolio and channel view:

• The next-generation cardiovascular therapeutics market report tracks the late-stage pipeline across lipids, heart failure, hypertension and thrombosis.
• The cardiovascular precision medicine market study covers genetic and biomarker-guided treatment, including Lp(a) testing and genotype-directed cardiomyopathy therapy.
• The specialty cardiovascular drugs market report focuses on high-cost specialty products distributed through specialty pharmacy.
• The cardiovascular specialty pharmaceuticals market study provides the company and portfolio view of the same segment.
• The cardiovascular prescription drugs market report covers the full prescription category, including the large generic base that specialty launches must displace.

3. Neuroscience and CNS: A Category Back in Growth

3.1 Alzheimer's disease and brain delivery

Lecanemab (Leqembi, full FDA approval in 2023) and donanemab (Kisunla, 2024) are the first therapies shown to slow Alzheimer's disease progression by clearing amyloid. Four reports cover the field:

• The Alzheimer's disease anti-amyloid antibody market report covers the class, including uptake constraints from diagnosis, infusion capacity and imaging monitoring for amyloid-related side effects.
• The Alzheimer's disease modifying drugs market study widens the lens to all disease-modifying mechanisms, including tau and neuroinflammation.
• The disease-modifying Alzheimer's disease therapeutics market report models the pipeline and the effect of blood-based diagnostics on the treated population.
• The dementia specialty pharmaceuticals market study covers symptomatic and behavioral treatments across dementia types.

Getting large molecules across the blood-brain barrier is the key enabling technology. Brain-shuttle antibodies that hitch a ride on the transferrin receptor, such as Roche's trontinemab, entered Phase 3 in 2025. Our next-generation brain shuttle therapeutics market report covers these platforms. The neurodegenerative disease biologics market and neuroscience biologic medicines market studies cover antibodies and other biologics across Alzheimer's, Parkinson's, ALS and multiple sclerosis, at the disease and the portfolio level respectively.

3.2 Psychiatry, pain and novel mechanisms

Psychiatry is seeing its first genuinely new mechanisms in decades:

• Cobenfy (xanomeline-trospium), a muscarinic agonist for schizophrenia, was approved in 2024 after Bristol Myers Squibb's roughly $14 billion acquisition of Karuna.
• Zuranolone brought a two-week oral treatment for postpartum depression in 2023.
• Suzetrigine, approved in January 2025, is the first non-opioid pain medicine with a new mechanism in over two decades.

Four reports cover this renewal:

• The novel CNS mechanism drugs market report tracks these first-in-class launches and the pipeline behind them.
• The mood disorder treatment drugs market study covers depression and bipolar disorder, including rapid-acting agents.
• The psychiatric prescription drugs market report sizes the full psychiatric category, where generics dominate volume.
• The long-acting injectable CNS drugs market study covers monthly to six-monthly antipsychotic injections that address adherence, the single largest cause of relapse in schizophrenia.

The rest of the CNS portfolio is covered by five reports:

• The neuroimmune modulators market for pain and neurodegeneration report covers drugs that target the inflammatory component of chronic pain and neurodegeneration.
• The CNS small molecule therapeutics market study covers the small-molecule segment, still the majority of CNS prescriptions.
• The high-complexity neurology therapies market report focuses on rare and specialist-managed neurological diseases.
• For portfolio sizing, the neurology and CNS therapeutics market and central nervous system therapeutics market reports provide category-level views by disease and by drug class respectively.
• The neuroscience specialty drugs market study covers the specialty channel.

4. Rare and Genetic Disease

4.1 Gene editing moves from ex vivo to in vivo

Casgevy (exagamglogene autotemcel) became the first approved CRISPR therapy in December 2023 for sickle cell disease and in January 2024 for beta-thalassemia. Four reports cover the first generation of editing therapies:

• Our CRISPR gene editing therapies market report covers the approved product and pipeline.
• The sickle cell gene editing therapies market study focuses on sickle cell disease, where ex vivo editing requires chemotherapy conditioning and specialist transplant centers.
• The gene editing for hemoglobinopathies market report extends coverage to beta-thalassemia and competing gene-addition therapies.
• The gene therapy clinical products market study tracks approved and late-stage gene therapies across all rare diseases, including recent safety and manufacturing challenges in the category.

The strategic frontier is in vivo editing, where the editor is delivered directly to the liver or other organs, usually by lipid nanoparticles. In 2025 a personalized base-editing therapy was used to treat an infant with a rare urea-cycle disorder, showing how quickly bespoke editing can now be designed. Three reports cover this shift:

• Our in vivo gene editing therapeutics market report covers the delivery platforms and clinical programs.
• The in vivo CRISPR base and prime editing therapeutics market study focuses on the newer editors that change single DNA letters without cutting both strands.
• The RNA splicing modulators market report covers an adjacent genetic approach proven by nusinersen and oral risdiplam in spinal muscular atrophy.

4.2 Cell replacement and type 1 diabetes

Replacing lost cells is the second genetic-medicine frontier, and type 1 diabetes is the lead indication. Donislecel (Lantidra) became the first approved allogeneic islet therapy in 2023, and stem-cell-derived islets have produced insulin independence in early trials. Five reports cover the area:

• The type 1 diabetes cell therapies market report covers these programs.
• The immune-evasive cell therapies market study covers gene-edited "hypoimmune" cells designed to survive without lifelong immunosuppression.
• The encapsulated cell replacement therapies market report covers devices that physically shield transplanted cells.
• The cell therapy for endocrine disorders market study extends the concept beyond diabetes.
• The allogeneic stem cell therapy market report covers off-the-shelf donor-derived products across indications.

4.3 CFTR, enzymes and the orphan portfolio

Cystic fibrosis shows how far small molecules can go in a genetic disease. Trikafta transformed outcomes after 2019, and a once-daily successor triple combination was approved in December 2024. Three reports cover the modulator classes:

• The CFTR modulator combination therapies market report covers the combinations.
• The next-generation CFTR correctors market study covers the corrector pipeline.
• The CFTR amplifier therapies market report covers amplifiers and genetic approaches for the roughly one in ten patients whose mutations modulators cannot help.

Enzyme replacement is the longest-established rare-disease model. Two reports cover it:

• The recombinant enzyme replacement therapies market report covers Gaucher, Fabry, Pompe and mucopolysaccharidoses, including next-generation enzymes with improved tissue uptake.
• The rare disease enzyme therapies market study adds substrate-reduction and chaperone approaches that compete with or complement enzyme replacement.

Five reports organize rare disease by disease area:

• The rare metabolic disorder drugs market report covers inborn errors of metabolism.
• The rare neuromuscular disease therapies market study covers SMA, Duchenne muscular dystrophy and myasthenic syndromes.
• The rare renal disease therapies market report covers IgA nephropathy, where endothelin, complement and APRIL-targeted drugs launched in quick succession.
• The rare liver disease therapeutics market study covers primary biliary cholangitis, where two PPAR agonists were approved in 2024.
• The rare respiratory diseases market report covers alpha-1 antitrypsin deficiency, bronchiectasis and other orphan lung conditions.

Seven further reports cover the orphan portfolio by modality:

• The rare disease biologics market and next-generation orphan biologics market reports cover the biologic segment, by approved portfolio and by pipeline.
• The rare genetic disease antibody therapies market and antibody-mediated rare disease therapies market studies cover antibodies that treat genetic diseases and rare diseases caused by pathogenic antibodies, respectively.
• The rare disease targeted drugs market report covers precision small molecules in orphan indications.
• The rare genetic disease drugs market study sizes the full genetic-disease category across modalities.

5. Infectious Disease, Antivirals and Vaccines

HIV is the clearest case of long-acting therapy reshaping a market. Cabotegravir-based injectables introduced every-one-to-two-month treatment and prevention in 2021. Lenacapavir, the first capsid inhibitor, was approved in 2025 as a twice-yearly injection for HIV prevention. Seven reports cover the field:

• Our HIV treatment and prevention drugs market report sizes the full category.
• The long-acting antiretroviral therapies market study models the shift from daily pills to injectables.
• The capsid inhibitor antiviral drugs market report covers the new mechanism class.
• The HIV maturation inhibitor drugs market study covers another mechanism being developed for long-acting combinations.
• The next-generation antiretroviral formulations market report covers implants, weekly oral regimens and other delivery innovations.

Hepatitis B is the next functional-cure target. Current antivirals suppress the virus but rarely clear it. Five reports cover the pipeline:

• The next-generation HBV therapeutics market report covers antisense, siRNA and immune-based candidates, led by bepirovirsen in Phase 3.
• The HBV core protein allosteric modulators market study covers a mechanism that disrupts viral assembly.
• The chronic hepatitis antiviral drugs market report sizes the hepatitis B and C antiviral market, including the curative hepatitis C regimens.
• The immune modulating agents for chronic viral infection market study covers approaches that restore the immune response needed for durable control.
• The chronic viral infection immune control therapies market report extends this to therapeutic vaccines and checkpoint approaches.

Four reports cover the wider infectious-disease market:

• The high-valent pneumococcal conjugate vaccine market report tracks the race to broader serotype coverage, from 20-valent and 21-valent vaccines approved since 2021 to 31-valent candidates in development.
• The antibody cocktail infectious disease therapies market study covers monoclonal antibody combinations for viral infections, a category shaped by COVID-19, Ebola and RSV.
• The anti-infective drugs market report sizes antibiotics, antifungals and antivirals, where antimicrobial resistance and weak commercial incentives remain structural problems.
• The AI-enhanced bacteriophage therapy market study covers AI-selected and engineered phages as a response to drug-resistant infections.

6. Plasma-Derived and Hemophilia Therapies

Plasma-derived medicines are a distinctive business: supply depends on collecting human plasma, and the United States supplies the majority of the world's source plasma. Four reports cover the segment:

• Our plasma-derived immunoglobulin therapies market report covers immunoglobulin, the largest plasma product, where demand growth in immunodeficiency and neurology consistently tests collection capacity.
• The immunoglobulin subcutaneous therapies market study covers the shift to home-administered subcutaneous products.
• The plasma-derived albumin therapies market report covers albumin, an important co-product whose demand is concentrated in China.
• The plasma-based coagulation factor therapies market study covers clotting factors for hemophilia and von Willebrand disease.

Plasma-derived and recombinant factors now compete with non-factor therapies. Emicizumab established prophylaxis with infrequent subcutaneous dosing, and anti-TFPI antibodies and an antithrombin-lowering siRNA were approved between 2024 and 2025. Our non-factor hemophilia therapeutics market report tracks how these products, together with gene therapy, are reshaping hemophilia treatment.

7. Bone, Renal, Respiratory, Urology, Women's Health and Aging

Several specialty areas are being reopened by new mechanisms or outcomes data:

• Bone. The bone health and osteoporosis drugs market report covers antiresorptive and anabolic therapy, including denosumab biosimilars since 2024. The sclerostin inhibitor bone therapies market and next-generation bone anabolic agents market studies focus on bone-building mechanisms led by romosozumab.
• Kidney. The nephrology specialty pharmaceuticals market report covers chronic kidney disease, where SGLT2 inhibitors, finerenone and GLP-1 outcomes data have created a multi-drug standard of care.
• Respiratory and sleep. The idiopathic pulmonary fibrosis (IPF) therapeutics market report covers antifibrotics and the late-stage pipeline. The breathing disorders treatment market study includes obstructive sleep apnea, where tirzepatide became the first approved drug in December 2024.
• Gout. The next-generation gout therapeutics market report covers urate-lowering therapy and treatments for uncontrolled disease.
• Urology. The urology therapeutics market study covers overactive bladder, benign prostatic hyperplasia and interstitial cystitis.
• Women's health. The women's health pharmaceuticals market report covers menopause, endometriosis, contraception and postpartum depression. Non-hormonal menopause treatment (fezolinetant, 2023) opened a new category.

Aging biology is the most speculative adjacency, but one attracting substantial private investment:

• The senolytics and age-related rejuvenation therapeutics market report covers drugs that clear senescent cells or restore tissue function.
• The senescence-targeting therapeutics market study focuses on specific senescence pathways.
• The next-gen pharmaceutical antioxidants and aging therapeutics market report covers mitochondrial and oxidative-stress approaches.

Where Value Is Created: The Specialty Pharma Value Chain

Regional perspective

• United States. The largest market and the primary launch market. Obesity reimbursement, Medicare negotiation and outcomes-based contracts for one-time therapies are the key policy variables.
• Europe. Stricter health technology assessment slows uptake of high-cost therapies such as anti-amyloid antibodies, while biosimilar and generic adoption is faster.
• China. A fast-growing developer of incretin and metabolic assets and a significant licensing source; domestic uptake depends on national reimbursement listing.
• Japan and emerging markets. Japan is an early adopter of Alzheimer's antibodies. Emerging markets drive generic volume and will be the next battleground for off-patent semaglutide.

What to Watch Through 2032

1. Oral and monthly obesity launches: uptake and payer coverage beyond diabetes will set the ceiling for the incretin market.
2. Lp(a) outcomes trials: a positive result would create a new preventive cardiology market almost overnight.
3. Blood-based Alzheimer's diagnostics: simpler diagnosis could expand anti-amyloid treatment far beyond today's specialist-center volumes.
4. In vivo editing safety and durability: these results will decide whether gene editing scales from rare disease to common conditions.
5. Twice-yearly HIV prevention uptake: a test of whether long-acting prevention can change epidemic trajectories.
6. Semaglutide patent expiry in several markets: from 2026, generic entry in major emerging markets will reshape pricing.

Frequently Asked Questions

What is driving growth in specialty pharmaceuticals outside oncology?

The main drivers are new mechanisms that address very large populations, particularly incretin-based obesity drugs, plus new cardiovascular, Alzheimer's and psychiatric therapies, genetic medicines for rare diseases and long-acting formats for HIV and schizophrenia.

What comes after Wegovy and Zepbound in obesity treatment?

The next generation includes oral GLP-1 drugs, multi-agonists that add glucagon or amylin activity, monthly or less frequent injectables and combination therapies that preserve muscle mass during weight loss.

Why is lipoprotein(a) an important cardiovascular target?

Elevated Lp(a) is an inherited cardiovascular risk factor affecting about one in five people, and it is not lowered by statins. Several antisense, siRNA and oral therapies are in outcomes trials, and none is yet approved.

How do anti-amyloid antibodies work in Alzheimer's disease?

Lecanemab and donanemab bind and clear amyloid plaques in the brain and have been shown to slow cognitive decline in early Alzheimer's disease. Their uptake depends on diagnosis, infusion capacity and MRI monitoring for side effects.

What is the difference between ex vivo and in vivo gene editing?

Ex vivo editing modifies a patient's cells outside the body and reinfuses them, often after chemotherapy conditioning, as with Casgevy. In vivo editing delivers the editing machinery directly into the body, usually by lipid nanoparticles, which could make treatment simpler and more scalable.

How are long-acting drugs changing HIV treatment and prevention?

Injectable cabotegravir and lenacapavir allow treatment or prevention every one to six months instead of daily pills. This improves adherence and is expected to shift a growing share of the HIV market to long-acting products.

Why is plasma supply important for immunoglobulin therapies?

Immunoglobulin is made from donated human plasma, so supply depends on collection capacity rather than factory output alone. Demand growth in immunodeficiency and neurological disease has repeatedly tested global plasma supply.

What do Mobility Foresights specialty pharma reports include?

Each linked report provides market size and multi-year forecasts, growth drivers and challenges, segmentation by product, indication and region, competitive landscape and company profiles, pipeline analysis and recent developments. Customization is available on request.

Work With Mobility Foresights

Mobility Foresights provides syndicated and custom market intelligence across metabolic, cardiovascular, neuroscience, rare disease and infectious disease markets. Each report linked above can be purchased individually or tailored to a specific indication, region or competitive question. Related analysis is available in our oncology and hematology therapeutics landscape. To request a sample or commission a bespoke study, contact our healthcare research team.