FDA Drug Approval Decisions

Last updated 31 July 2026. Covering target action dates from August to December 2026.

The closing stretch of the third quarter has become the busiest regulatory window of 2026. Between early August and the end of September, the US Food and Drug Administration faces target action dates spanning oncology, haematology, rare neurology, gene therapy and diagnostic imaging. The September 2026 FDA decisions carry particular weight, because several involve candidates that would become the first therapy ever approved for their indication.

That calendar arrives on the back of an eventful July. The agency cleared four products between 10 and 24 July, three of them ahead of schedule. In the final week of the month, FDA reviewers put sharply critical assessments in front of two advisory committees on consecutive days, and the panels reached opposite conclusions. Both decisions land in August.

Upcoming decisions

Recently decided

On 10 July the FDA approved pembrolizumab, or pembrolizumab with berahyaluronidase alfa-pmph, each combined with enfortumab vedotin, as neoadjuvant treatment followed by adjuvant treatment after cystectomy for adults with muscle-invasive bladder cancer. The decision extends an existing approval from cisplatin-ineligible patients to all patients who are candidates for cystectomy, and arrived more than five weeks before its 17 August target date. Clearance rested on the Phase 3 EV-304/KEYNOTE-B15 trial.

Four days later the agency approved gedatolisib (Revtorpyk; Celcuity) with fulvestrant, with or without palbociclib, for adults with hormone receptor-positive, HER2-negative advanced breast cancer without a detected PIK3CA mutation. Efficacy was established in Study 1 of the Phase 3 VIKTORIA-1 trial, which enrolled 392 patients. Celcuity says Revtorpyk is the first and only approved therapy to inhibit all four Class I PI3K isoforms alongside mTORC1 and mTORC2.

On 22 July the agency approved zidesamtinib (Jideytro) for adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer who have received a prior ROS1 kinase inhibitor. That approval arrived almost two months before its 18 September target date, and was announced by GSK, which had completed a $10.6 billion acquisition of Nuvalent on 15 July. Clearance rested on the single-arm Phase 1/2 ARROS-1 trial, in which 117 previously treated patients recorded a confirmed objective response rate of 44% by blinded independent central review. Among those who had received only one prior ROS1 inhibitor the rate was 49%, falling to 38% in patients who had received two or more.

Two days after that, Otsuka Pharmaceutical secured approval for centanafadine (Simtriyo), a once-daily extended-release capsule for ADHD in adults and children aged six and over weighing at least 20kg. The approval rests on four randomised, double-blind, placebo-controlled Phase 3 trials. Otsuka describes it as the first and only approved norepinephrine, dopamine and serotonin reuptake inhibitor.

RP1 in advanced melanoma: a panel vote reverses the mood before 2 August

Replimune’s oncolytic immunotherapy RP1 (vusolimogene oderparepvec), given with nivolumab, has a goal date of 2 August 2026 for advanced melanoma that has progressed on anti-PD-1 therapy. It is the company’s third attempt. The FDA issued complete response letters in July 2025 and April 2026. The April letter centred on whether the single-arm design of the Phase 1/2 IGNYTE trial could isolate the contribution of RP1 when it is combined with nivolumab, and also cited heterogeneity in the study population, uncertainty in response assessment including potentially confounding surgical intervention, and the limited randomised dataset submitted from IGNYTE-3. The agency accepted the latest resubmission on 26 June as a complete, class 1 response, a classification indicating that the deficiencies were addressed with existing data rather than new studies.

In IGNYTE, 140 patients with confirmed progression on an anti-PD-1 regimen achieved a blinded independent central review objective response rate of 33.6% by modified RECIST, with a median duration of response of 24.8 months and 44.8% of responders still in response at three years. Updated three-year data presented at the 2026 ASCO Annual Meeting showed median overall survival of 32.9 months. Just under half of all treated patients, 47.8%, were alive at three years, rising to 83.5% among responders.

Briefing documents published ahead of the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee restated the objection running through both previous rejections. Because RP1 is injected directly into tumours and was studied without a nivolumab-only control arm, reviewers argued the data could not separate a systemic anti-tumour effect from a local one. They also questioned the statistical approach used to derive the response rate. Replimune shares fell roughly 30% on publication.

The committee did not agree. On 30 July it voted 10 to 3 that the efficacy results from IGNYTE are evaluable and clinically meaningful. More than 30 speakers, including patients who had received the therapy, supported approval during the open public hearing. Hussein Tawbi of MD Anderson Cancer Center argued the therapy should be available to patients until the Phase 3 trial reads out. Among the dissenters, Paul Chapman of Weill Cornell Medicine said he had nothing to compare the single-arm results against.

The vote is advisory and non-binding, and the FDA is not obliged to follow it, though the agency generally does. With the decision due on 2 August, RP1 enters its action date in a considerably stronger position than the briefing documents implied 48 hours earlier.

Deramiocel: a negative panel vote before 22 August

Capricor Therapeutics’ deramiocel, an allogeneic cardiosphere-derived cell therapy for Duchenne muscular dystrophy, went before the same advisory committee on 29 July ahead of a 22 August target date. The panel voted three for and nine against, with no abstentions, that the available evidence did not support effectiveness in Duchenne-related cardiomyopathy.

Two features of the meeting matter for how the vote should be read. The voting question covered a narrower indication than Capricor had proposed, and the committee was not asked to vote on the overall benefit-risk profile. In a separate discussion of upper limb function, feedback was directionally supportive of the Phase 3 HOPE-3 evidence, including the primary endpoint PUL 2.0. Capricor is seeking approval for both upper limb impairment and cardiomyopathy in patients aged nine and over.

The meeting was contentious. Chief executive Linda Marbán opened by saying parts of the FDA briefing document were hard to reconcile with what had actually happened. The dispute concerns which statistical analysis plan governs the results: the agency drew on an earlier prespecified plan, while Capricor maintains that version 3.0, finalised before unblinding, is the controlling document. Panellists also raised missing data, sensitivity analyses and heterogeneity in the cardiac outcomes.

The application is supported by the Phase 2 HOPE-2 trial and its open-label extension alongside HOPE-3, which met both its primary and secondary endpoints when reported in December 2025. Deramiocel received a complete response letter in July 2025 citing inadequate evidence of effectiveness and unresolved chemistry, manufacturing and controls items. The vote does not bind the agency, but Capricor shares fell sharply and analysts moved towards expecting a second complete response letter rather than approval.

Zanidatamab moves to the front line: 25 August

Jazz Pharmaceuticals faces a 25 August target action date for Ziihera (zanidatamab-hrii) with chemotherapy, with or without the PD-1 inhibitor tislelizumab, as first-line treatment for HER2-positive locally advanced or metastatic gastro-oesophageal adenocarcinoma. The bispecific HER2-directed antibody already holds accelerated approval in previously treated biliary tract cancer.

The supplemental application rests on HERIZON-GEA-01, a global Phase 3 trial of 914 previously untreated patients, published in The New England Journal of Medicine in May 2026. Both zanidatamab arms cut the risk of progression or death by roughly 35% against trastuzumab plus chemotherapy. Median overall survival reached 26.4 months in the triplet arm against 19.2 months in the control arm, a 28% reduction in the risk of death (hazard ratio 0.72). The zanidatamab and chemotherapy doublet reached a median overall survival of 24.4 months but did not meet the prespecified threshold for statistical significance at this interim analysis (hazard ratio 0.80), and a further analysis is expected.

The trial was led by Kohei Shitara of the National Cancer Center Hospital East in Japan, with Elena Elimova of the Princess Margaret Cancer Centre in Toronto among the co-authors. Investigators reported it as the first Phase 3 trial in metastatic gastro-oesophageal adenocarcinoma to show median progression-free survival beyond one year and median overall survival beyond two years.

Ropeginterferon in essential thrombocythaemia: 30 August

PharmaEssentia has a 30 August target date for a label expansion of Besremi (ropeginterferon alfa-2b-njft) into adult essential thrombocythaemia, a myeloproliferative neoplasm characterised by excessive platelet production and an elevated risk of clotting. The application is supported by the global Phase 3 SURPASS-ET trial against anagrelide, with confirmatory evidence from the Phase 2b EXCEED-ET study.

In SURPASS-ET, conducted in high-risk patients resistant or intolerant to hydroxyurea, Besremi produced a durable clinical response rate of 42.9% against 6.0% for anagrelide (p=0.0001). Taiwan approved the same expansion in June 2026, the first regulatory clearance of the product in this indication anywhere in the world. The company describes it as the first new therapy approved for essential thrombocythaemia in close to 30 years.

The rest of the August cluster

August also carries several dates held over from the July window. Moderna’s mRNA-1010 influenza vaccine is due on 5 August. Bristol Myers Squibb’s iberdomide, with daratumumab and dexamethasone, in relapsed or refractory multiple myeloma has a 17 August date. Ultragenyx’s DTX401 gene therapy for glycogen storage disease type Ia follows on 23 August, the subcutaneous lecanemab starting dose on 24 August, Gilead’s bictegravir and lenacapavir single tablet on 27 August, and ITM’s 177Lu-edotreotide for gastroenteropancreatic neuroendocrine tumours on 28 August.

September 2026 FDA decisions in rare disease

Three of the September decisions concern conditions with no approved treatment at all.

Ultragenyx’s UX111 (rebisufligene etisparvovec), an AAV9 gene therapy for Sanfilippo syndrome type A, has a 19 September date. The FDA issued a complete response letter in July 2025 over chemistry, manufacturing and controls information and observations from facility inspections, then accepted the resubmission in April 2026 The filing draws on the Phase 1/2/3 Transpher A study and includes up to eight years of follow-up. In a subgroup of 17 patients treated under the age of two or at an earlier disease stage, the Bayley-III cognitive raw score treatment effect against natural history reached 23.2 points (p<0.0001). Emil Kakkis, chief executive of Ultragenyx, has framed the review as a step towards what would be the first therapy approved for the condition.

Ionis Pharmaceuticals has a 22 September date for zilganersen, an antisense oligonucleotide designed to reduce production of glial fibrillary acidic protein in Alexander disease, a rare astrocytopathy. The pivotal study, which Ionis describes as Phase 1-3 and which is registered with ClinicalTrials.gov as Phase 3, enrolled 54 participants aged 1.5 to 53 years across 13 sites in eight countries. It met its primary endpoint, showing stabilisation of gait speed on the 10-metre walk test at week 61 in participants aged five and over (least squares mean difference 33.3%, p=0.041). Data presented at AAN 2026 covered 53 participants aged 2 to 53. Zilganersen holds breakthrough therapy, orphan drug and rare paediatric disease designations.

Zilurgisertib, an oral ALK2 inhibitor licensed by Mirum Pharmaceuticals from Incyte, has a 26 September date for fibrodysplasia ossificans progressiva in patients aged 12 and over. The disease, in which soft tissue progressively turns to bone, affects around 300 people in the United States and 900 worldwide. Cohort 1 of the placebo-controlled Phase 2 PROGRESS study, reported at ENDO 2026, showed reductions in total heterotopic ossification lesion volume, in new lesions and in flare activity.

Winrevair, glioma imaging and a date that slipped

Not every September decision involves a first-in-disease therapy. Merck has a 21 September target date for a further label update to Winrevair (sotatercept-csrk) in pulmonary arterial hypertension. The FDA accepted the supplemental application in February 2026. It rests on the Phase 3 HYPERION study in adults diagnosed within the previous 12 months, which reported a 76% reduction in the risk of clinical worsening events against placebo (hazard ratio 0.24, 95% confidence interval 0.14 to 0.41). A separate label update based on the Phase 3 ZENITH trial was approved in October 2025, so this is the second expansion of the product in under a year.

The earliest September date belongs to diagnostics. Telix Pharmaceuticals has an 11 September date for Pixclara (floretyrosine F 18), a PET agent for distinguishing recurrent or progressive glioma from treatment-related change. The agency issued a complete response letter in 2025 requesting further confirmatory diagnostic performance data, and Telix resubmitted in March 2026. Telix says no amino acid PET agent for brain tumour imaging is currently approved in the United States, although several international guidelines recommend the class.

One September date has already moved. Praxis Precision Medicines announced on 29 June that the FDA had extended its review of relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies from 27 September to 27 December, after additional sensitivity analyses were classified as a major amendment. No new clinical studies were requested and no safety or manufacturing concerns were cited.

What the calendar signals

Two patterns run through this window.

The first is speed. Four July approvals arrived on or before their deadlines, one by nearly two months. Where the evidence has been randomised and the endpoints uncontested, review teams have been closing files ahead of the PDUFA framework rather than against it.

The second is harder to read. RP1, deramiocel, UX111 and Pixclara are all resubmissions following complete response letters, and the agency has reopened each file rather than send the sponsor back to the clinic. In the last week of July it put comprehensively critical assessments in front of two advisory committees on consecutive days. The panels then split: nine to three against deramiocel, ten to three in favour of RP1.

The distinction between the two is instructive. Both disputes were methodological rather than clinical, but they were not the same methodological dispute. The RP1 question was what a single-arm trial can establish in a population with few remaining options, and the panel decided that a 33.6% response rate with a median duration beyond two years was interpretable enough to act on. The deramiocel question was narrower and more technical: which statistical analysis plan governs the result, and whether the cardiac endpoints held up under sensitivity analysis. Panels appear willing to tolerate an imperfect trial design where the effect is durable and the alternative is nothing. They appear less willing to tolerate uncertainty about how a result was derived.

Both files also arrived through the same door. The complete response letters for RP1 and deramiocel were issued while Vinay Prasad led the Center for Biologics Evaluation and Research, a tenure that drew sustained criticism from industry and rare disease advocates for the handling of single-arm and externally controlled evidence, and support from those who favoured higher evidentiary standards. Prasad left the agency at the end of April 2026. Commissioner Marty Makary resigned on 12 May after 13 months in post, and Kyle Diamantas, previously deputy commissioner for food, was named acting commissioner. Diamantas is a lawyer rather than a physician, and the administration has had difficulty securing Senate confirmation for health appointments, so the interim arrangement may run for some time.

It would be premature to read a single favourable panel vote as a change of direction. Advisory committees are not the agency, the FDA is not bound by their recommendations, and the deramiocel vote points the other way. The August decisions on RP1 and deramiocel will say considerably more than the votes themselves. What can be said is that published deadlines have held, with relutrigine the only date in this window to have moved, and for procedural reasons.

The September cluster is weighted towards rare disease filings where randomisation is difficult and natural history comparisons do much of the work, which is precisely the territory these disputes have concerned. For patients with Sanfilippo syndrome type A, Alexander disease and fibrodysplasia ossificans progressiva, those dates represent something rarer than a regulatory milestone: the prospect of a first approved treatment.