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Every verified treatment and trial we have published, newest first. Filter by country, access level, or patient stories.

Once-Daily Pill for Adults with Untreated CLL or SLL

Jaypirca (pirtobrutinib) — once-daily pill for adults with previously untreated CLL or SLL and no known 17p deletion

US label section 1.3 added 10/2026; BRUIN CLL-313 NCT05023980

This is for adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have not been treated before and have no known 17p deletion.

FDA ApprovedUnited States

Heart Pill for Obstructive HCM from About 66 Pounds

Camzyos (mavacamten) — oral treatment for symptomatic obstructive HCM from about 66 lb

US label expanded 9/2026 to patients ≥30 kg including eligible adolescents; SCOUT-HCM NCT06253221; REMS required

This is for families living with symptomatic obstructive hypertrophic cardiomyopathy when the person weighs at least 30 kg (about 66 lb).

Pill for Teens and Adults with Severe Alopecia Areata

Olumiant (baricitinib) — once-daily pill for teens and adults with severe alopecia areata

US label expanded 09/2026 to ages 12+; EU SmPC already covers adolescents; BRAVE-AA-PEDS NCT05723198

This is for families and adults living with severe alopecia areata who are 12 years or older.

FDA ApprovedUnited States

Infusion to Reduce Relapses in Childhood Nephrotic Syndrome

Gazyva (obinutuzumab) — infusion to reduce relapse risk in childhood-onset idiopathic nephrotic syndrome

The United States approved Gazyva (obinutuzumab) on 25 September 2026 to reduce relapses of childhood-onset nephrotic syndrome.

This is for families and adults living with frequently relapsing or steroid-dependent childhood-onset idiopathic nephrotic syndrome who are in complete…

Once-Weekly Oral Medicine for Kids’ BRAF Brain Tumors

Ojemda (tovorafenib) — once-weekly oral RAF inhibitor for BRAF-altered pediatric low-grade glioma

Ojemda (tovorafenib) is a weekly medicine for children’s low-grade glioma with certain BRAF gene changes, after earlier treatment.

This is for children and young people — and caregivers — facing relapsed or refractory pediatric low-grade glioma with a BRAF fusion, rearrangement, or V600…

FDA ApprovedUnited States

Once-Daily Pill for Adults with Parkinson’s Disease

Juvmo (tavapadon) — selective D1/D5 dopamine agonist for Parkinson’s disease in adults

Juvmo (tavapadon) is a once-daily pill for adults with Parkinson’s disease.

This is for adults with Parkinson’s disease — and caregivers — who need a labelled once-daily oral option that can be used with or without levodopa.

FDA ApprovedUnited States

Monthly Shot to Lower Triglycerides and Pancreatitis Risk

Tryngolza (olezarsen) — ApoC-III ASO for severe hypertriglyceridemia (sHTG) and FCS

Tryngolza (olezarsen) is a monthly shot. In June 2026, the United States approved it for adults with severe high triglycerides.

This is for adults with severe hypertriglyceridemia (fasting TG 500 mg/dL or higher) — and caregivers — who need a labelled way to lower triglycerides and cut…

FDA ApprovedUnited States

Once-Daily Pill for FOP Bone Growth in Teens and Adults

Atebrioz (zilurgisertib) — oral ALK2 inhibitor to reduce new heterotopic ossification in FOP (ages 12+)

Atebrioz (zilurgisertib) is a once-daily pill for fibrodysplasia ossificans progressiva in people 12 and older.

This is for teens 12 and older and adults with fibrodysplasia ossificans progressiva (FOP) — and caregivers — who need a labelled way to slow new extra bone…

FDA ApprovedUnited States

New Oral Pill Option for Previously Treated Bile Duct Cancer

Lyrfigtu (lirafugratinib) — oral FGFR2 inhibitor for previously treated FGFR2-altered cholangiocarcinoma

The United States approved Lyrfigtu (lirafugratinib) on 23 September 2026. It is for adults with bile duct cancer that cannot be removed or has spread, with an…

This is for adults with previously treated unresectable, locally advanced, or metastatic bile duct cancer (cholangiocarcinoma) that harbors an FGFR2 gene…

Once-Weekly Basal Insulin Shot for Adults with Type 2 Diabetes

Onswik (insulin efsitora alfa-gobe) — once-weekly basal insulin for adults with type 2 diabetes

Onswik (insulin efsitora alfa) is a once-weekly long-acting insulin for adults with type 2 diabetes.

This is for adults with type 2 diabetes — and caregivers helping them — who may be candidates for a once-weekly basal insulin instead of a daily basal shot.

FDA ApprovedUnited States

New Prescription Ointment for Mild-to-Moderate Eczema Ages 2 and Up

Adquey (difamilast) — topical ointment for mild-to-moderate eczema (atopic dermatitis)

FDA approved Adquey (difamilast) ointment, 1% on 12 February 2026 (NDA 219474) for topical treatment of mild to moderate atopic dermatitis in adults and…

This is for people with mild to moderate atopic dermatitis (eczema) — and caregivers of children 2 years and older — who may be candidates for labelled US…

First FDA-Approved Treatment for MCT8 Peripheral Thyrotoxicosis

Emcitate (tiratricol) — dispersible tablets for peripheral thyrotoxicosis in MCT8 deficiency

FDA approved Emcitate (tiratricol) tablets for oral suspension on 28 September 2026 — Novel Drug Approvals 2026 #45 — for treatment of peripheral…

This is for people living with MCT8 deficiency (Allan-Herndon-Dudley syndrome) — and the caregivers who help them — who need a realistic route to tiratricol.

FDA ApprovedUnited States

Ameluz Red-Light PDT Now FDA-Approved for Superficial Basal Cell Skin Cancer

Ameluz (aminolevulinic acid HCl 10% gel) — red-light PDT for superficial basal cell carcinoma

FDA labelled expansion on 9 September 2026 (sNDA 208081/S-040; Prescribing Information Revised 09/2026) for Ameluz (aminolevulinic acid hydrochloride) topical…

This is for adults with superficial basal cell carcinoma — and the caregivers helping them — who may be candidates for labelled US Ameluz red-light PDT with a…

FDA ApprovedUnited States

First FDA-Approved Implant Treatment for MacTel Eye Disease

Encelto (revakinagene taroretcel-lwey) — encapsulated cell implant for macular telangiectasia type 2

FDA approval on 5 March 2025 for Encelto (revakinagene taroretcel-lwey) implant — an allogeneic encapsulated cell-based gene therapy for intravitreal surgical…

This is for adults living with idiopathic macular telangiectasia type 2 — and the caregivers who help them — who may be candidates for labelled US Encelto…

FDA ApprovedUnited States

First FDA-Approved Treatment for Ataxia in Ataxia-Telangiectasia

Aqneursa (levacetylleucine) — oral suspension for ataxia in ataxia-telangiectasia

FDA labelled expansion on 18 September 2026 for Aqneursa (levacetylleucine) for oral suspension — for the treatment of ataxia in adult and pediatric patients…

This is for people living with ataxia-telangiectasia — and the caregivers who help them — who weigh at least 15 kg and may be candidates for labelled US…

FDA ApprovedUnited States

First FDA-Approved Gene Therapy for AADC Deficiency

Kebilidi (eladocagene exuparvovec-tneq) — one-time intraputaminal gene therapy for aromatic L-amino acid decarboxylase deficiency

FDA accelerated approval on 13 November 2024 (press 14 November 2024) for Kebilidi (eladocagene exuparvovec-tneq) suspension — an AAV vector-based gene therapy…

This is for families of children (and adults) with genetically confirmed AADC deficiency from biallelic DDC changes who have achieved skull maturity and may be…

FDA ApprovedUnited States

First FDA-Approved Gene Therapy Sheets for RDEB Wounds

Zevaskyn (prademagene zamikeracel) — gene-modified cellular sheets surgically applied to treat wounds in recessive dystrophic epidermolysis bullosa

FDA approval on 28 April 2025 for Zevaskyn (prademagene zamikeracel) gene-modified cellular sheets — an autologous cell sheet-based gene therapy for…

This is for people living with recessive dystrophic epidermolysis bullosa — and the caregivers who help them — who have chronic open wounds and may be…

FDA ApprovedUnited States

First FDA-Approved Treatment for Adults With Recurrent Respiratory Papillomatosis

Papzimeos (zopapogene imadenovec-drba) — four subcutaneous shots over 12 weeks for adults with recurrent respiratory papillomatosis

FDA traditional/full approval on 14 August 2025 for Papzimeos (zopapogene imadenovec-drba) suspension — a non-replicating adenoviral vector immunotherapy given…

This is for adults living with recurrent respiratory papillomatosis who still need repeated airway surgeries and may be candidates for labelled US Papzimeos…

FDA ApprovedUnited States

First FDA-Approved Gene Therapy for Wiskott-Aldrich Syndrome

Waskyra (etuvetidigene autotemcel) — one-time gene-modified stem-cell therapy for Wiskott-Aldrich syndrome when no matched related donor is available

FDA approval on 9 December 2025 for Waskyra (etuvetidigene autotemcel) suspension — an autologous hematopoietic stem-cell gene therapy given as a one-time IV…

This is for families of children (and adults) with genetically confirmed Wiskott-Aldrich syndrome who need a transplant-style path and do not have a suitable…

FDA ApprovedUnited States

First FDA-Approved Gene Therapy for Sanfilippo Syndrome Type A

Fayuvi (rebisufligene etisparvovec-hopf) — one-time IV gene therapy for children with Sanfilippo syndrome type A (MPS IIIA)

FDA approval on 17 September 2026 for Fayuvi (rebisufligene etisparvovec-hopf) suspension — a one-time AAV9 gene therapy given by intravenous infusion — for…

This is for families of children with Sanfilippo syndrome type A (MPS IIIA) who still have preserved neurodevelopmental function and may be candidates for…

FDA ApprovedUnited States

New Pill Option for Kidney Disease in Adults With Type 1 Diabetes

Kerendia (finerenone) — once-daily oral pill to lower urine protein in adults with kidney disease and type 1 diabetes

FDA supplemental approval announced 17 September 2026 for a new use of Kerendia (finerenone) tablets — a once-daily oral non-steroidal mineralocorticoid…

This is for adults with type 1 diabetes who already have chronic kidney disease with protein in the urine and may be candidates for labelled US Kerendia as a…

FDA ApprovedUnited States

First FDA-Approved Pill for Children 4–11 With APDS

Joenja (leniolisib) — first labelled oral treatment for children 4–11 with APDS who weigh at least 27 kg

FDA supplemental approval on 11 September 2026 for Joenja (leniolisib) tablets — expanding the approved APDS pathway to adult and pediatric patients 4 years of…

This is for caregivers of children 4–11 years with genetically confirmed APDS who weigh at least 27 kg (about 60 lb) and who may be candidates for labelled US…

FDA ApprovedUnited States

New Muscle-Targeted IV Treatment for SMA Ages 2 and Up

Isembyld (apitegromab-mstn) — a once-every-4-weeks IV medicine that targets muscle for people with SMA already on an SMN2 treatment

FDA approval on 11 September 2026 for Isembyld (apitegromab-mstn) injection — a muscle-targeted intravenous medicine — for spinal muscular atrophy (SMA) in…

This is for children and adults living with SMA who are already on an SMN2-targeted treatment (such as nusinersen or risdiplam) and who may be candidates for…

FDA ApprovedUnited States

New Heart-Protection Use for Mounjaro in Type 2 Diabetes

Mounjaro (tirzepatide) — weekly shot for adults with type 2 diabetes that now also carries a labelled heart-protection use

FDA supplemental approval effective 27 August 2026 for a new use of Mounjaro (tirzepatide) injection — a once-weekly subcutaneous dual GIP/GLP-1 receptor…

This is for adults with type 2 diabetes who are at high risk for heart attack, stroke, or cardiovascular death, and who may already be candidates for weekly…

FDA ApprovedUnited States

New Once-Daily Pill for Adults With HIV on Complex Treatment

Bixlenvo (bictegravir and lenacapavir) — a once-daily two-drug pill for adults with HIV who are already undetectable, including people on complex multi-tablet regimens

FDA approval on 27 August 2026 for Bixlenvo (bictegravir 75 mg / lenacapavir 50 mg) tablets — a once-daily complete regimen — to replace the current…

This is for adults living with HIV who are already undetectable on a stable regimen — including people taking several pills a day or complex multi-drug…

FDA ApprovedUnited States

New Radiation Treatment for Hormone-Sensitive Metastatic Prostate Cancer

Pluvicto (lutetium Lu 177 vipivotide tetraxetan) — targeted radiation given with hormone-pathway pills for metastatic prostate cancer that still responds to hormone therapy

FDA approval on 31 July 2026 for a new use of Pluvicto (lutetium Lu 177 vipivotide tetraxetan) injection — a radioligand given by IV — in combination with…

This is for adults with metastatic prostate cancer that still responds to hormone-pathway therapy, whose tumors light up on a PSMA PET scan, and who may be…

FDA ApprovedUnited States

First FDA-Approved Cell Therapy to Reduce Chronic Graft-Versus-Host Disease

TREGZI (allogeneic regulatory T cell immunotherapy with HSPC and T cells-vldq) — donor cell transplant product to help adults with blood cancers stay free of chronic graft-versus-host disease

FDA approval on 30 June 2026 for TREGZI (allogeneic regulatory T cell immunotherapy with HSPC and T cells-vldq) suspension — a donor-matched cell therapy given…

This is for adults with blood cancers who need a matched-donor stem cell transplant after intensive conditioning and who may be candidates for labelled US…

FDA ApprovedUnited States

First FDA-Approved Gene Therapy for Severe Leukocyte Adhesion Deficiency

KRESLADI (marnetegragene autotemcel) — one-time gene therapy for children with severe leukocyte adhesion deficiency type I when no matched sibling donor is available

FDA accelerated approval on 26 March 2026 for KRESLADI (marnetegragene autotemcel) suspension — an autologous hematopoietic stem cell-based gene therapy for…

This is for families of children with severe LAD-I from confirmed ITGB2 gene changes who do not have an HLA-matched sibling donor and who may be candidates for…

FDA ApprovedUnited States

New Tumor Injection for Advanced Melanoma After Immunotherapy Progresses

TUDRIQEV (vusolimogene oderparepvec-wtpg) — engineered virus injected into tumors plus nivolumab for unresectable advanced cutaneous melanoma after PD-1 therapy progress

FDA accelerated approval on 6 August 2026 for TUDRIQEV (vusolimogene oderparepvec-wtpg) suspension — a genetically modified oncolytic viral therapy for…

This is for adults with unresectable advanced cutaneous melanoma whose cancer progressed on a PD-1 immunotherapy and who may be candidates for labelled US…

FDA ApprovedUnited States

First FDA-Approved Gene Therapy for Glycogen Storage Disease Type Ia

GENGLYCOS (pariglasgene brecaparvovec-opnr) — one-time gene therapy to help lower daily cornstarch needs in glycogen storage disease type Ia (ages 8 and older)

FDA accelerated approval on 19 August 2026 for GENGLYCOS (pariglasgene brecaparvovec-opnr) suspension — a one-time AAV8 gene therapy given by intravenous…

This is for people 8 years and older with glycogen storage disease type Ia who still rely on carefully timed cornstarch and may be candidates for labelled US…

FDA ApprovedUnited States

New Weekly Shot for Protein Leak from IgA Kidney Disease

Trutakna (atacicept-vymj) — weekly under-the-skin shot to lower protein in the urine in adults with primary IgA nephropathy

FDA accelerated approval on 7 July 2026 for Trutakna (atacicept-vymj) injection — a weekly subcutaneous medicine — to reduce proteinuria in adults with primary…

This is for adults with primary IgA nephropathy who leak protein into the urine and may be candidates for labelled US Trutakna once-weekly home shots.

First FDA-Approved Eye Form of Bevacizumab for Wet AMD

Lytenava (bevacizumab-vikg) — ophthalmic VEGF inhibitor injection for wet age-related macular degeneration

FDA approval on 24 July 2026 for Lytenava (bevacizumab-vikg) injection — an ophthalmic VEGF inhibitor for intravitreal use — for neovascular (wet) age-related…

This is for adults with wet age-related macular degeneration who may be candidates for labelled US Lytenava monthly eye injections — the first FDA-approved…

FDA ApprovedUnited States

First FDA-Approved Gene Therapy for Genetic Hearing Loss

OTARMENI (lunsotogene parvec-cwha) — gene therapy for severe genetic hearing loss from biallelic OTOF variants

FDA accelerated approval on 23 April 2026 for OTARMENI (lunsotogene parvec-cwha) suspension — a one-time intracochlear gene therapy — for pediatric and adult…

This is for children and adults with severe genetic hearing loss from confirmed OTOF gene changes who may be candidates for labelled US OTARMENI gene therapy…

FDA ApprovedUnited States

A New Copper Treatment for Children with Menkes Disease

Zycubo (copper histidinate) — subcutaneous copper replacement for Menkes disease in pediatric patients

FDA approval on 12 January 2026 for Zycubo (copper histidinate) for injection — a subcutaneous copper replacement — for Menkes disease in pediatric patients.

This is for families of children with Menkes disease who may be candidates for labelled US Zycubo subcutaneous copper replacement — especially when treatment…

FDA ApprovedUnited States

New Treatment for Immune-Related Anemia in Teens and Adults

Imaavy (nipocalimab-aahu) — neonatal Fc receptor blocker for warm autoimmune hemolytic anemia in adults and adolescents 12 years and older after corticosteroid exposure

FDA approval on 25 August 2026 for Imaavy (nipocalimab-aahu) injection — a neonatal Fc receptor (FcRn) blocker — for warm autoimmune hemolytic anemia (wAIHA)…

This is for people 12 years and older with warm autoimmune hemolytic anemia who are currently on or have previously received corticosteroids and who may be…

Health Canada/FDA ApprovedUnited StatesCanadaJapanEuropean Union

New Pill for Hormone-Positive Advanced Breast Cancer

Etcamah (camizestrant) — oral ER antagonist with CDK4/6 for ESR1m HR+/HER2− advanced breast cancer

FDA accelerated approval on 4 September 2026 for Etcamah (camizestrant) tablets — an oral estrogen receptor antagonist — in combination with a CDK4/6 inhibitor…

This is for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer who are already on an aromatase inhibitor plus a CDK4/6…

FDA ApprovedUnited States

New Treatment for a Rare Blood Cancer (BPDCN) in Adults

Decnupaz (pivekimab sunirine-pvzy) — CD123 ADC for adults with BPDCN

FDA approval on 27 May 2026 for Decnupaz (pivekimab sunirine-pvzy) — a CD123-directed antibody and alkylating-agent conjugate — for adults with blastic…

This is for adults with BPDCN — an ultra-rare, aggressive blood/bone-marrow cancer that often involves skin, marrow, nodes, and other organs — who may be…

FDA ApprovedUnited States

Weekly Treatment for Children with Achondroplasia

Yuviwel (navepegritide) — once-weekly CNP analog for children ≥2 with achondroplasia

FDA accelerated approval on 27 February 2026 for Yuviwel (navepegritide) for injection — a C-type natriuretic peptide (CNP) analog / TransCon CNP prodrug — to…

This is for families of children aged 2 years and older with achondroplasia and open growth plates who may be candidates for a labelled US once-weekly…

FDA ApprovedUnited States

New Pill for Ovarian Cancer That No Longer Responds to Chemo

Lifyorli (relacorilant) — oral GR antagonist with nab-paclitaxel for platinum-resistant ovarian cancer

FDA traditional (full) approval on 25 March 2026 for Lifyorli (relacorilant) capsules — an oral glucocorticoid receptor (GR) antagonist — in combination with…

This is for adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer after one to three prior systemic regimens…

FDA ApprovedUnited StatesChina

New Pill for Mantle Cell Lymphoma After Prior Treatment

Beqalzi (sonrotoclax) — oral BCL-2 inhibitor for R/R mantle cell lymphoma after ≥2 lines including a BTK inhibitor

FDA accelerated approval on 13 May 2026 for Beqalzi (sonrotoclax) tablets — an oral BCL-2 inhibitor — for adults with relapsed or refractory mantle cell…

This is for adults with relapsed or refractory mantle cell lymphoma who have already had at least two systemic treatments, including a BTK inhibitor, and who…

FDA ApprovedUnited States

New IV Treatment for Advanced Hormone-Positive Breast Cancer

Revtorpyk (gedatolisib) — IV pan-PI3K/mTOR inhibitor for HR+/HER2−, PIK3CA-wild-type advanced breast cancer

On 14 July 2026 the FDA labelled Revtorpyk (gedatolisib) for injection — an intravenous kinase inhibitor of class I PI3K isoforms (α, β, δ, γ) and…

Revtorpyk is a US-labelled IV pan-PI3K / mTOR inhibitor used with fulvestrant, with or without palbociclib, for adults whose HR+/HER2− advanced breast cancer…

FDA ApprovedUnited States

New Oral Treatment for Multiple Myeloma After Prior Therapy

Zenbexus (iberdomide) — first labelled CELMoD for adults with multiple myeloma after ≥1 prior line

FDA accelerated approval on 13 August 2026 for Zenbexus (iberdomide) capsules — an oral cereblon-modulating protein degrader (CELMoD) — in combination with…

This is for adults with multiple myeloma who have already had at least one prior treatment that included a proteasome inhibitor and an immunomodulatory drug…

FDA ApprovedUnited States

New Pill for a Rare Gene-Related Lung Cancer

Jideytro (zidesamtinib) — oral ROS1 kinase inhibitor after a prior ROS1 TKI

On 22 July 2026 the FDA labelled Jideytro (zidesamtinib) tablets for adults with locally advanced or metastatic ROS1-positive NSCLC who received a prior ROS1…

Jideytro is a US-labelled oral pill for adults whose ROS1-positive lung cancer has already been treated with another ROS1 kinase inhibitor.

FDA ApprovedUnited StatesJapan

New Oral Medicine to Help Prevent COVID-19 After Exposure

Xocova / Zokovea (ensitrelvir) — oral COVID-19 post-exposure prophylaxis

The first FDA-labelled oral option for COVID-19 post-exposure prophylaxis (PEP).

Xocova (ensitrelvir) is the first pill approved in the United States to help prevent COVID-19 after contact with someone who has it.

FDA ApprovedUnited States

New Infusion When Soft Tissue Turns to Bone (Adult FOP)

Pasatru (garetosmab-grts) for adults with fibrodysplasia ossificans progressiva (FOP)

The FDA labelled an intravenous activin A–blocking monoclonal antibody on 19 August 2026 to reduce formation of new heterotopic ossification (HO) lesions and…

Pasatru (garetosmab) is an infusion every 4 weeks for adults with fibrodysplasia ossificans progressiva.

Health Canada/FDA ApprovedUnited StatesCanada

New Weekly Enzyme Treatment for Arginase-1 Deficiency

Loargys (pegzilarginase-nbln) for arginase-1 deficiency (ARG1-D)

The FDA (accelerated, 23 Feb 2026) and Health Canada (full NOC, 29 May 2026) have labelled a weekly enzyme replacement for hyperargininemia in people 2 years…

Loargys (pegzilarginase) is a weekly enzyme treatment for arginase 1 deficiency in people 2 and older, used with a low-protein diet.

FDA ApprovedUnited States

New Oral Antibiotic for Complicated Urinary Tract Infections

Utebzi (tebipenem pivoxil) — first oral carbapenem for adults with complicated UTI

The first FDA-approved oral carbapenem for adults.

Utebzi (tebipenem pivoxil) is the first carbapenem antibiotic pill approved in the United States.

FDA ApprovedUnited States

New Daily Pill for Advanced Breast Cancer After Hormone Therapy

Veppanu (vepdegestrant) — first FDA-approved PROTAC for ESR1-mutated ER+/HER2− advanced breast cancer

The FDA labelled a once-daily 200 mg oral PROTAC estrogen-receptor degrader tablet — take with food, swallow whole — for adults with ER-positive…

Veppanu (vepdegestrant) is a daily pill.

FDA ApprovedUnited States

New Weekly Infusion for Children with Hunter Syndrome

Avlayah (tividenofusp alfa-eknm) for neurologic Hunter syndrome (MPS II)

FDA accelerated approval (24 March 2026 letter; press 25 March 2026) for Avlayah (tividenofusp alfa-eknm) — a once-weekly IV enzyme replacement for neurologic…

Avlayah is a weekly infusion for the brain effects of Hunter syndrome in children of at least 5 kilograms.

FDA ApprovedUnited States

New Infusion Treatment for Thyroid Eye Disease

Lumvoa (veligrotug-vvze) for thyroid eye disease (active and chronic)

The FDA labelled an intravenous IGF-1R monoclonal antibody for thyroid eye disease regardless of disease activity or duration on 26 June 2026. The labelled…

Lumvoa (veligrotug) is an infusion for thyroid eye disease, whether the disease is active or long-standing.

FDA ApprovedUnited States

New Pill for Hard-to-Control High Blood Pressure

Baxfendy (baxdrostat) — first aldosterone-synthase inhibitor for adults whose blood pressure is not adequately controlled

The FDA labelled a once-daily 1 mg or 2 mg oral aldosterone synthase inhibitor tablet — take with or without food, swallow whole — in combination with other…

Baxfendy (baxdrostat) is a once-daily pill for adults whose high blood pressure is not controlled on other medicines.

FDA ApprovedUnited States

New Daily Pill for Moderate-to-Severe Psoriasis (Ages 12+)

Icotyde (icotrokinra) for moderate-to-severe plaque psoriasis — 12 and older, at least 40 kg

The FDA labelled a once-daily 200 mg oral IL-23 receptor peptide for moderate-to-severe plaque psoriasis in people 12 and older who weigh at least 40 kg and…

Icotyde (icotrokinra) is a once-daily pill for moderate to severe plaque psoriasis.

Once-Weekly Insulin Shot for Adults with Type 2 Diabetes

Awiqli (insulin icodec / insulin icodec-abae) — once-weekly basal insulin

The first labelled once-weekly basal insulin.

Awiqli (insulin icodec) is a once-weekly long-acting insulin. The United States approved it in March 2026 only for adults with type 2 diabetes.

New Pill for Chronic Hives Not Controlled by Antihistamines

Rhapsido (remibrutinib) for adult chronic spontaneous urticaria still active on H1 antihistamines

The FDA, Health Canada, the European Commission, and the UK MHRA have labelled an oral BTK inhibitor — 25 mg twice daily — for adults with chronic spontaneous…

Rhapsido (remibrutinib) is a twice-daily pill for adults whose chronic hives continue despite antihistamines (United States, Canada, Europe, United Kingdom).

FDA ApprovedUnited States

New Daily Pill to Lower High ("Bad") Cholesterol

Lipfendra (enlicitide) — first oral PCSK9 inhibitor for high LDL-C in adults

The FDA labelled a once-daily 20 mg oral PCSK9 inhibitor tablet — take in the morning on an empty stomach — as an adjunct to diet and exercise to lower LDL-C…

Lipfendra (enlicitide) is a daily pill taken on an empty stomach. The United States approved it, with diet and exercise, to lower “bad” cholesterol in adults.

FDA ApprovedUnited States

First FDA-Approved Treatment for Alexander Disease

Zanvastro (zilganersen) for Alexander disease (AxD)

The first FDA-labelled medicine for Alexander disease — an ultra-rare, progressive GFAP leukodystrophy.

Zanvastro (zilganersen) is the first approved medicine for Alexander disease, a very rare brain disease.

Health Canada/FDA ApprovedUnited StatesCanadaEuropean Union

First FDA-Approved Treatment for Chronic Hepatitis D

Hepcludex (bulevirtide) for chronic hepatitis D (HDV)

The first labelled drug for chronic hepatitis D. The United States (FDA accelerated, 22 May 2026) labels 8.5 mg subcutaneously once daily for adults without…

Hepcludex (bulevirtide) is the first approved medicine for chronic hepatitis D. The United States gave accelerated approval to a daily 8.5 mg shot in May 2026.…

New Oral Medicine for Narcolepsy Type 1 in Adults

Orzeyful (oveporexton) for narcolepsy type 1

The FDA labelled oveporexton for adults with narcolepsy type 1 on 5 August 2026 — and said it will be lawful to market only after DEA scheduling.

Orzeyful (oveporexton) is approved in the United States for adults with narcolepsy type 1. It cannot be sold there until the Drug Enforcement Administration…

Twice-Yearly Shot for a Severe Type of Asthma

Exdensur (depemokimab) for severe eosinophilic asthma — and, outside the United States, for nasal polyps

The FDA, Health Canada, the UK MHRA, Japan’s MHLW, the EU Commission, and (on the Australian PI) the TGA have labelled a twice-yearly anti-IL-5 injection.

Exdensur (depemokimab) is a shot given twice a year for severe eosinophilic asthma. In the United States, it is approved for this asthma in people 12 and older.

Weekly Shot When Too Many Red Blood Cells Need Frequent Draws

Mimrylo (rusfertide) for erythrocytosis in adults with polycythemia vera (PV)

The FDA labelled a once-weekly subcutaneous hepcidin mimetic for adults with polycythemia vera who still need frequent phlebotomies despite standard care.

Health Canada/FDA ApprovedUnited StatesCanada

Once-Daily Switch Pill for Adults with Suppressed HIV

Idvynso (doravirine/islatravir) for HIV-1 — switch-only dual tablet

Health Canada and the FDA labelled a once-daily doravirine/islatravir tablet as a switch option for adults with HIV-1 who are already suppressed — not a…

Idvynso (doravirine and islatravir) is a daily pill for adults whose HIV (human immunodeficiency virus) is already under control.

New Daily Pill for Chronic Weight Management in Adults

Foundayo (orforglipron) for chronic weight management

FDA labelled oral orforglipron for chronic weight management in adults. UK MHRA also labelled weight management and type 2 diabetes — those lines stay separate.

New Daily Pill for Adult Muscle and Skin Inflammation

Lisraya (brepocitinib) — first oral TYK2/JAK1 tablet labelled for adult dermatomyositis

FDA labelled a once-daily 30 mg pill for dermatomyositis in adults (27 August 2026), with a boxed JAK-class warning.

New Pill for Itch from a Bile-Duct Disease

Lynavoy (linerixibat) for cholestatic itch in adults with primary biliary cholangitis — oral IBAT inhibitor

The FDA (17 Mar 2026) and UK MHRA (1 May 2026) labelled an oral 40 mg tablet twice daily for cholestatic pruritus associated with primary biliary cholangitis…

Lynavoy (linerixibat) is a pill taken twice daily for itch caused by primary biliary cholangitis, a liver disease, in adults.

Health Canada/FDA ApprovedUnited StatesCanada

New Pill for a Gene-Related Advanced Lung Cancer

Hyrnuo (sevabertinib) for HER2 TKD-mutant NSCLC

Hyrnuo (sevabertinib) is a pill for adults with advanced lung cancer and a certain HER2 gene mutation.

New Pill for Advanced Pancreatic Cancer

Rasonque (daraxonrasib): the first RAS(ON) pill for metastatic pancreatic adenocarcinoma

The FDA approved this RAS(ON) pill on 26 August 2026 for metastatic pancreatic adenocarcinoma. It is not licensed in Canada.

The FDA approved this RAS(ON) pill on 26 August 2026 for metastatic pancreatic adenocarcinoma. It is not a Canadian pharmacy drug.

New Pill for Scarred-Lung Disease in Adults

Jascayd (nerandomilast) for IPF and PPF

The FDA, Europe, and the UK have labelled nerandomilast for adults with idiopathic pulmonary fibrosis and for adults with progressive pulmonary fibrosis.

Jascayd (nerandomilast) is approved in the United States, Europe and the United Kingdom.

Health Canada/FDA ApprovedUnited StatesCanadaEuropean Union

Injection Treatment When the Body Makes Too Many Eosinophils

Fasenra (benralizumab) for hypereosinophilic syndrome

Health Canada and the FDA have labelled Fasenra for hypereosinophilic syndrome. Japan and Switzerland have too. The UK SmPC already carries the wording.

New Pill When the Heart Muscle Is Too Thick

Myqorzo (aficamten) for obstructive hypertrophic cardiomyopathy

The US, Europe, the UK and China have labelled aficamten for adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM).

Myqorzo (aficamten) is approved in the United States, Europe, the United Kingdom, China and Canada for adults with symptoms from obstructive hypertrophic…