Featured
FDA New Drug
Approvals
2026
13
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Pierre Fabre / Atara
Tabelecleucel (Ebvallo)
EBV+ Post-Transplant Lymphoproliferative Disease
CRL
Aquestive Therapeutics
Anaphylm (dibutepinephrine)
Anaphylaxis
CRL
REGENXBIO
RGX-121 (clemidsogene)
Hunter Syndrome (MPS II)
CRL
Aldeyra Therapeutics
Reproxalap
Dry Eye Disease
CRL
Vanda Pharmaceuticals
Tradipitant
Gastroparesis
CRL
FDA Season 2026 — Scorecard
Pierre Fabre / Atara
Tabelecleucel (Ebvallo)
EBV+ Post-Transplant Lymphoproliferative Disease
Aquestive Therapeutics
Anaphylm (dibutepinephrine)
Anaphylaxis
REGENXBIO
RGX-121 (clemidsogene)
Hunter Syndrome (MPS II)
Aldeyra Therapeutics
Reproxalap
Dry Eye Disease
Vanda Pharmaceuticals
Tradipitant
Gastroparesis
Exclusive Interviews
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Arbor Biotechnologies sets out CNS gene editing push with three ALS candidates at Meeting on the Mesa
CEO Devyn Smith presented preclinical data for gene editing programs targeting C9orf72, STMN2 and UNC13A, with a first CNS IND filing targeted for 2027.
Jason Jones, Head of Global Business Development, Cellular Origins | Onyx Live | ISCT Dublin 2026
Cell and gene therapy can get treatments approved – scaling them is the hard part. Cellular Origins' Jason Jones on automating manufacturing to reach 100,000 patients a year.
Prostate screening could be some men’s only NHS touchpoint in years. With public role models shifting stigma, it should be treated as early detection and a route back into routine care.
The story of how an Indian biotech startup slashed the cost of CAR-T from $475,000 to just $30,000 and started a revolution in global CAR-T therapy access.
“Preventative care spending fell from 14.1% to just 5.2% of Government healthcare spending.” Ben Phillips warns that welfare reforms could widen existing health gaps.
Anis Fahandej-Sadi breaks down the big problems with current incentive structures
"Almost a third of over-65s will fall each year," but could nGVS represent a discreet tool against imbalance?
To live in the grey zone of sub-clinical pathology is to be unwell enough to hurt, but not enough to be heard.
First-generation medical students carry two textbooks - one devoted to anatomy, the other towards cultivating resilience.
Trillions of microbes inside us are not mere bystanders, but quiet architects of our moods.
"These projects are more than acts of goodwill," China's Health Silk Road is as much about soft power as it is about medicine.
“Pain is treated like a badge of honor" in medicine, but the cost is a workforce breaking under invisible pressure.
FDA approvals - March 2026
New options emerged in PBC-associated itch, plaque psoriasis, neurologic Hunter syndrome, and platinum-resistant ovarian cancer.
Novo Nordisk's Awiqli delivers the first once-weekly basal insulin for type 2 diabetes
The first once-weekly basal insulin reaches US patients, cutting injection burden from 365 to 52 per year and demonstrating superior glycaemic control in the ONWARDS program
FDA approves Rocket's Kresladi as the first gene therapy for severe leukocyte adhesion deficiency-I
The first gene therapy for severe LAD-I achieves 100% HSCT-free survival with sustained CD18 expression through 42 months in paediatric patients lacking a matched donor
Denali’s AVLAYAH wins FDA accelerated approval in Hunter syndrome
The accelerated approval in Hunter syndrome was supported by Phase 1/2 biomarker data and adds a Priority Review Voucher alongside Denali’s first approved blood-brain barrier-penetrant biologic.
FDA Clears Corcept’s Relacorilant Plus Nab-Paclitaxel in Platinum-Resistant Ovarian Cancer
Corcept’s FDA approval establishes relacorilant as the first FDA-approved selective glucocorticoid receptor antagonist
Johnson & Johnson’s ICOTYDE brings a new oral IL-23 option to plaque psoriasis
On March 17, 2026, the FDA approved Johnson & Johnson’s ICOTYDE (icotrokinra) for adults and pediatric patients 12 years and older who weigh at least 40 kg with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. ICOTYDE is a once-daily oral IL-23
FDA approves Alfasigma’s Lynavoy for primary biliary cholangitis
On March 17, 2026, the US FDA approved Lynavoy (linerixibat) for cholestatic pruritus associated with primary biliary cholangitis. The approval came days after GSK and Alfasigma announced a March 9 licensing agreement under which Alfasigma acquired exclusive worldwide rights to develop, manufacture, and commercialize linerixibat. Under the agreement, GSK receives
FDA greenlights Ascendis’ once-weekly YUVIWEL to boost growth in children with achondroplasia
On February 27, 2026, the FDA approved Ascendis Pharma’s YUVIWEL (navepegritide) (ASND, Nasdaq, Close: $233.50 - as of Feb. 27, 2026) to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. Navepegritide is a once-weekly subcutaneous C-type natriuretic peptide
Immedica Pharma’s Loargys adds an enzyme-replacement entrant for ultra-rare ARG1-D–related hyperargininemia
On February 23, 2026, the FDA approved Loargys (pegzilarginase-nbln) from Immedica Pharma for hyperargininemia in adults and pediatric patients 2 years and older with Arginase 1 Deficiency, in conjunction with dietary protein restriction. ARG1-D affects an estimated 250 people in the U.S.A. Immedica Pharma’s CEO,
FDA approves Vanda’s Bysanti for schizophrenia and bipolar I manic or mixed episodes
On February 20, 2026, the FDA approved Bysanti (milsaperidone) from Vanda Pharmaceuticals (VNDA, Nasdaq) for schizophrenia and for manic or mixed episodes associated with bipolar I disorder in adults. Vanda describes Bysanti as a new chemical entity atypical antipsychotic that is bioequivalent to iloperidone across the therapeutic dosing spectrum, enabling
Acrotech Biopharma brings a new topical PDE4 option - Adquey - into a crowded eczema market.
On February 12, 2026, the FDA approved Adquey (difamilast 1%) ointment from Acrotech Biopharma for the topical treatment of mild to moderate atopic dermatitis in adults and pediatric patients aged 2 years and older. The commercial question centers on where difamilast can earn durable share against entrenched topicals. Mechanistically, difamilast
Kim Brown, CEO, Compass Biomedical | Onyx Live | ISCT Dublin 2026
Everyone watches the final cell therapy – far fewer watch what the cells are grown in. Compass Biomedical's Kim Brown on why culture media quietly shapes the product.
Jason Jones, Head of Global Business Development, Cellular Origins | Onyx Live | ISCT Dublin 2026
Cell and gene therapy can get treatments approved – scaling them is the hard part. Cellular Origins' Jason Jones on automating manufacturing to reach 100,000 patients a year.
Dimitrios Kouroupis, Professor, University of Miami | Onyx Live | ISCT Dublin 2026
Exosomes are "cell therapy without the cells" – and University of Miami's Dimitrios Kouroupis sees these cell-free vesicles reshaping musculoskeletal medicine.
Jon Ellis, CEO, Trenchant BioSystems | Onyx Live | ISCT Dublin 2026
Fewer than 10% of eligible CAR-T patients ever get treated. Trenchant BioSystems CEO Jon Ellis says the bottleneck is no longer the science – it's manufacturing.
Sean Werner, CTO, BioLife Solutions | Onyx Live | ISCT Dublin 2026
Most cell therapies arrive at the patient frozen – and BioLife Solutions CTO Sean Werner says the field still treats what happens after manufacturing as an afterthought.
Augustin de Bettignies, Chief Business Officer, MPC Therapeutics | Onyx Live | ISCT Dublin 2026
Most of cell therapy fixates on the cell's target. MPC Therapeutics' Augustin de Bettignies says its metabolism — what it burns during manufacturing — matters just as much.
Clene Nanomedicine's ALS drug is a gold nanocrystal you drink. It missed its primary endpoint. The FDA is still listening.
CEO Rob Etherington and chief scientist Mark Mortenson on the bioenergetic thesis behind CNM-Au8, the HEALEY data, and five meetings with the agency.
The sugar layer biotech forgot - and why Anomer Bio thinks small molecules can finally drug it
Co-founder and CEO Sophie James on modified monosaccharides, intracellular glycan processing, and a rare-disease-first route into fibrosis, oncology, and Alzheimer's.
Slone Partners’ Leslie Loveless on the post-boom talent reset in biopharma
Biopharma’s boom-era org charts are giving way to leaner, faster operating models. Slone Partners CEO, Leslie Loveless, says the edge now goes to pragmatic builders: “The companies that win right now are hiring leaders who can do more with less.”
A world-expert in Stargardt disease has developed a pill to slow vision loss: why Tinlarebant could win FDA approval
After years studying Stargardt’s natural history, Belite Bio’s Dr. Hendrik Scholl is championing Tinlarebant as a practical early intervention - “Intervention is simply one pill a day - and we are able to slow down the progression of disease by more than one-third.”
Belite Bio concludes phase 3 Stargardt trials with nearly 40% retinal cell preservation
Belite is positioning Tinlarebant for regulatory submission after Phase 3 Stargardt data, emphasizing the long-term safety burden of a systemic, lifelong oral therapy: “For a lifelong treatment, the safety bar is about as high as it gets.”
Why Flatiron is betting on data quality, not hype, to power the next era of cancer care
With global oncology scale and nearly 2,000 scientific publications, Flatiron Health is positioning itself as the industry’s most trusted evidence partner by pairing AI-driven speed with uncompromising scientific rigor. “We don’t chase hype. We build trust, and in healthcare that’s non-negotiable.”
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