International coverage of treatments and trials, with the ability to zoom in on countries we cover. From approved options to compassionate access.
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…
This is for families of children (and adults) with genetically confirmed AADC deficiency from biallelic…
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…
This is for people living with recessive dystrophic epidermolysis bullosa — and the caregivers who help them…
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…
This is for adults living with recurrent respiratory papillomatosis who still need repeated airway surgeries…
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…
This is for families of children (and adults) with genetically confirmed Wiskott-Aldrich syndrome who need 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…
This is for families of children with Sanfilippo syndrome type A (MPS IIIA) who still have preserved…
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…
This is for adults with type 1 diabetes who already have chronic kidney disease with protein in the urine and…
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 labelled APDS door to adult and pediatric patients 4…
This is for caregivers of children 4–11 years with genetically confirmed APDS who weigh at least 27…
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…
This is for children and adults living with SMA who are already on an SMN2-targeted treatment (such as…
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…
This is for adults with type 2 diabetes who are at high risk for heart attack, stroke, or cardiovascular…
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…
This is for adults living with HIV who are already undetectable on a stable regimen — including people taking…
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…
This is for adults with metastatic prostate cancer that still responds to hormone-pathway therapy, whose…
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…
This is for adults with blood cancers who need a matched-donor stem cell transplant after intensive…
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…
This is for families of children with severe LAD-I from confirmed ITGB2 gene changes who do not have an…
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…
This is for adults with unresectable advanced cutaneous melanoma whose cancer progressed on a PD-1…
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…
This is for people 8 years and older with glycogen storage disease type Ia who still rely on carefully timed…
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…
This is for adults with primary IgA nephropathy who leak protein into the urine and may be candidates for…
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…
This is for adults with wet age-related macular degeneration who may be candidates for labelled US Lytenava…
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…
This is for children and adults with severe genetic hearing loss from confirmed OTOF gene changes who may be…
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…
This is for families of children with Menkes disease who may be candidates for labelled US Zycubo…
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…
This is for people 12 years and older with warm autoimmune hemolytic anemia who are currently on or have…
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…
This is for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer who are…
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…
This is for adults with BPDCN — an ultra-rare, aggressive blood/bone-marrow cancer that often involves skin,…
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…
This is for families of children aged 2 years and older with achondroplasia and open growth plates who may be…
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…
This is for adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer…
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…
This is for adults with relapsed or refractory mantle cell lymphoma who have already had at least two…
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)…
Revtorpyk is a US-labelled IV pan-PI3K / mTOR inhibitor used with fulvestrant, with or without…
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…
This is for adults with multiple myeloma who have already had at least one prior treatment that included a…
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…
Jideytro is a US-labelled oral pill for adults whose ROS1-positive lung cancer has already been treated with…
Xocova / Zokovea (ensitrelvir) — oral COVID-19 post-exposure prophylaxis
The first FDA-labelled oral option for COVID-19 post-exposure prophylaxis (PEP). On 29 May 2026 the FDA approved Xocova (ensitrelvir) tablets…
The first FDA-labelled oral option for COVID-19 post-exposure prophylaxis (PEP). On 29 May 2026…
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)…
The FDA labelled an intravenous activin A–blocking monoclonal antibody on 19 August 2026 to reduce…
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…
The FDA (accelerated, 23 Feb 2026) and Health Canada (full NOC, 29 May 2026) have labelled a weekly enzyme…
Utebzi (tebipenem pivoxil) — first oral carbapenem for adults with complicated UTI
The first FDA-approved oral carbapenem for adults. On 17 June 2026 the FDA labelled Utebzi (tebipenem pivoxil) tablets for complicated urinary…
The first FDA-approved oral carbapenem for adults. On 17 June 2026 the FDA labelled Utebzi…
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,…
The FDA labelled a once-daily 200 mg oral PROTAC estrogen-receptor degrader tablet — take with food,…
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…
FDA accelerated approval (24 March 2026 letter; press 25 March 2026) for Avlayah (tividenofusp…
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…
The FDA labelled an intravenous IGF-1R monoclonal antibody for thyroid eye disease regardless of disease…
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…
The FDA labelled a once-daily 1 mg or 2 mg oral aldosterone synthase inhibitor tablet — take with or…
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…
The FDA labelled a once-daily 200 mg oral IL-23 receptor peptide for moderate-to-severe plaque psoriasis in…
Awiqli (insulin icodec / insulin icodec-abae) — once-weekly basal insulin
The first labelled once-weekly basal insulin. The United States (FDA, 26 Mar 2026) labels it only for adults with type 2 diabetes as an adjunct to diet…
The first labelled once-weekly basal insulin. The United States (FDA, 26 Mar 2026) labels it only for…
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…
The FDA, Health Canada, the European Commission, and the UK MHRA have labelled an oral BTK inhibitor — 25 mg…
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…
The FDA labelled a once-daily 20 mg oral PCSK9 inhibitor tablet — take in the morning on an empty…
Zanvastro (zilganersen) for Alexander disease (AxD)
The first FDA-labelled medicine for Alexander disease — an ultra-rare, progressive GFAP leukodystrophy. On 3 September 2026 the FDA approved Zanvastro…
The first FDA-labelled medicine for Alexander disease — an ultra-rare, progressive GFAP leukodystrophy. On…
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…
The first labelled drug for chronic hepatitis D. The United States (FDA accelerated, 22 May 2026) labels…
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. Japan’s PMDA…
The FDA labelled oveporexton for adults with narcolepsy type 1 on 5 August 2026 — and said it will be lawful…
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. The…
The FDA, Health Canada, the UK MHRA, Japan’s MHLW, the EU Commission, and (on the Australian PI) the TGA have…
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.…
The FDA labelled a once-weekly subcutaneous hepcidin mimetic for adults with polycythemia vera who still need…
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…
Health Canada and the FDA labelled a once-daily doravirine/islatravir tablet as a switch option for 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…
FDA labelled oral orforglipron for chronic weight management in adults. UK MHRA also labelled weight…
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…
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…
The FDA (17 Mar 2026) and UK MHRA (1 May 2026) labelled an oral 40 mg tablet twice daily for cholestatic…
Hyrnuo (sevabertinib) for HER2 TKD-mutant NSCLC
The FDA and Health Canada have labelled an oral HER2 tyrosine-kinase inhibitor for adults with locally advanced or metastatic NSCLC whose tumours have HER2…
The FDA and Health Canada have labelled an oral HER2 tyrosine-kinase inhibitor for adults with locally…
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 a Canadian pharmacy drug. SAP, a first-line trial…
The FDA approved this RAS(ON) pill on 26 August 2026 for metastatic pancreatic adenocarcinoma. It is not a…
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.…
The FDA, Europe, and the UK have labelled nerandomilast for adults with idiopathic pulmonary fibrosis and for…
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. The EU…
Health Canada and the FDA have labelled Fasenra for hypereosinophilic syndrome. Japan and Switzerland have…
Myqorzo (aficamten) for obstructive hypertrophic cardiomyopathy
The FDA, Europe, the UK, and China have labelled aficamten for symptomatic obstructive HCM in adults. England's NHS has a 30-day funding clock from NICE.…
The FDA, Europe, the UK, and China have labelled aficamten for symptomatic obstructive HCM in adults.…