Rare Disease FDA Approval Calendar

Priority review. Enspryng blocks the interleukin-6 receptor and is given as an under-the-skin injection, every 4 weeks in its approved NMOSD use, which would make it the first at-home subcutaneous treatment for thyroid eye disease, the autoimmune complication of Graves' disease that pushes the eyes forward and can cause double vision. In the Phase 3 SatraGO-2 trial, 53% of patients had their eye bulging (proptosis) improve by at least 2 mm at week 24 versus 23% on placebo; the twin SatraGO-1 trial showed 49% versus 31%, which did not reach statistical significance. Enspryng is already approved for NMOSD, and a separate application for MOGAD is due January 10, 2027.

DNA immunotherapy for adults with recurrent respiratory papillomatosis, given as 4 injections into muscle with INOVIO's CELLECTRA device. INOVIO seeks accelerated approval; the FDA accepted the application under standard review and flagged a preliminary view that eligibility for accelerated approval had not been justified. Would be the second RRP treatment after Papzimeos (August 2025).

Oral pill under priority review for accelerated approval; would be the first pyruvate kinase activator for sickle cell disease. The filing rests on the placebo-controlled RISE UP Phase 2 and Phase 3 trials in patients 16 and older. In Phase 3, mitapivat met its hemoglobin response endpoint, but its other primary endpoint, reducing sickle cell pain crises, did not reach statistical significance. The confirmatory REIGNITE trial is testing whether it reduces transfusion needs. Mitapivat is already approved for adults with PK deficiency (Pyrukynd, 2022) and adults with thalassemia (Aqvesme, December 2025).

The FDA moved the decision from August 17 to November 17, 2026 after classifying Beren's March 18, 2026 response to an information request as a major amendment. Adrabetadex is a cyclodextrin drug given into the spinal fluid. The application has priority review and Breakthrough Therapy and Orphan Drug designations.

Inhaled therapy; review period extended by 3 months.

Cell therapy for DMD. At a July 29, 2026 meeting, an FDA advisory committee voted 9 to 3 that the evidence did not show effectiveness for Duchenne cardiomyopathy, the use originally requested. Capricor then amended the application with 24-month data from the Phase 3 HOPE-3 trial and asked the FDA to consider a refined indication focused on upper limb function, the trial's primary endpoint. The FDA classified the amendment as major and extended the review 3 months, from August 22 to November 22.

Oral brain-penetrant therapy for the neurological form of Gaucher disease.

Oral ribitol therapy under priority review; LGMD2I/R9 has no approved treatment.

Dual PPAR agonist under priority review for second-line PBC.

Priority review. In the Phase 3 PEAK trial, adding bezuclastinib to sunitinib extended median progression-free survival from 9.2 to 16.5 months in patients whose GIST had progressed on imatinib, which Cogent describes as the first treatment to beat an active comparator in a GIST trial. The FDA said it does not plan an advisory committee.

Vertex seeks accelerated approval for adults with IgA nephropathy, with povetacicept given as an injection under the skin every 4 weeks. In a planned Week 36 interim analysis of the Phase 3 RAINIER trial, it lowered urine protein 49.8% more than placebo. Like Trutakna (atacicept), approved in July 2026, it blocks both BAFF and APRIL. Would join a fast-growing IgAN treatment field that had zero targeted therapies before December 2021.

IL-36 receptor antibody targeting generalized pustular psoriasis flares. Vanda's August 5, 2026 quarterly release refers to the product by the proposed brand name Quimilza and reaffirmed the December 12 target action date.

Resubmitted after a June 10, 2026 complete response letter, the second for this application, which cited findings from an inspection at a third-party manufacturing site and raised no efficacy or safety concerns. The FDA accepted the resubmission on July 17, 2026. Would be the first self-administered long-acting octreotide in the US; already approved in the EU as Oczyesa.

BTK inhibitor already approved in Japan for this rare brain lymphoma.

An enzyme from Streptococcus pyogenes, given as a single infusion hours before transplant, that cleaves IgG antibodies so a highly sensitized patient (cPRA 99.9% or higher, with a positive crossmatch) can receive a kidney that their immune system would otherwise reject immediately. These patients make up an estimated 10 to 15% of the transplant waiting list and can wait indefinitely on dialysis. Holds Fast Track and Orphan Drug designations; Hansa requested priority review, and the FDA later set a December 19, 2026 action date, 12 months after the December 2025 submission. In the 64-patient US Phase 3 ConfIdeS trial, average kidney function (eGFR) at 12 months was 51.5 with imlifidase versus 19.3 in the control arm, and significantly more patients were off dialysis. Would be the first US treatment for this group; conditionally approved in the EU and UK as Idefirix since 2020.

A one-time BCMA-directed CAR T cell therapy from Kite, a Gilead company, which bought Arcellx outright in April 2026. The application covers people whose myeloma has come back after at least 3 prior lines of treatment. In the pivotal iMMagine-1 study, 96% of 117 treated patients responded.

Review extended 3 months from the original September date.

Selective KIT D816V inhibitor for the most common form of systemic mastocytosis.

Resubmission for a rare metabolic disorder with no approved treatment.

Priority review. Would be the first approved treatment for MOGAD, a rare autoimmune disease that attacks the optic nerves, spinal cord and brain. In the Phase 3 METEOROID trial, Enspryng cut the risk of relapse by 68%, with 87% of treated patients relapse-free at 48 weeks versus 67% on placebo. Already approved for NMOSD.

Priority review for accelerated approval, based on the increase in dystrophin it produced. The exon 51 skipping drug is linked to an antibody fragment that binds transferrin receptor 1 to carry it into muscle, and it is given once every 4 weeks.

Priority review. Would convert the 2018 accelerated approval, which covers only refractory disease, to full approval, and Insmed says that could bring Arikayce to patients earlier in their disease course. The application rests on the Phase 3b ENCORE study in 425 adults with a new MAC lung infection, which met its main goal on respiratory symptoms; adding Arikayce to standard antibiotics also raised culture conversion by month 6 to 88% from 57%.

Follow-on filing to expand Joenja's September 2026 pediatric approval (ages 4-11, at least 27 kg) down to children weighing as little as 13 kg, using weight-based dosing. Supported by an open-label, multinational, single-arm Phase 3 study showing improvements in lymphadenopathy and naive B cell counts. Priority review.

Once-daily oral pill under priority review for children with achondroplasia, the most common form of dwarfism; it would be an alternative to the 2 approved injections, daily Voxzogo and weekly Yuviwel. In the Phase 3 PROPEL 3 trial, infigratinib raised annualized height velocity by about 1.74 cm per year over placebo in the main analysis, or 2.10 cm per year as a simple average. It has Orphan Drug, Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations.

Would be the first approved treatment for Stargardt disease.

Sarepta asked the FDA to convert the accelerated approvals of Amondys 45 and Vyondys 53 to traditional approvals, citing its ESSENCE confirmatory study and real-world evidence. Sarepta announced on June 30, 2026 that the FDA accepted both applications. Both drugs stay available under their accelerated approvals during the review.

Application to convert the 2021 accelerated approval into full approval.

Would be the first in vivo CRISPR gene editing therapy for hereditary angioedema, given as a single one-time infusion.

DR5 agonist antibody; would be the first approved systemic therapy for this rare bone cancer, which has no approved drug treatment today.

Oral on-demand and prophylactic bradykinin B2 antagonist.

Gamma secretase inhibitor for rare, locally aggressive soft-tissue tumors.

Norepinephrine reuptake inhibitor targeting cataplexy in narcolepsy.

A pill that turns down an overactive calcium-sensing receptor, the cause of ADH1. It is under standard review, and the FDA is not planning an advisory committee meeting. BridgeBio says it would be the first therapy approved specifically for ADH1.

Oral CELMoD pill taken with carfilzomib and dexamethasone, for myeloma that has come back after at least 1 prior treatment that included an anti-CD38 antibody and lenalidomide. Patients in the SUCCESSOR-2 trial had a median of 2 prior lines of therapy.

Self-injected antibody that dials down B cells without depleting them. In the Phase 3 INDIGO trial it cut the risk of IgG4-RD flare by 56% over 52 weeks, with 73% of treated patients flare-free versus 45% on placebo, and reduced steroid use. Zenas disclosed the acceptance and date in its August 13, 2026 quarterly update.

Once-daily oral prostacyclin receptor agonist.

Cogent's third bezuclastinib application, for the aggressive form of the disease. In the APEX trial, 65% of patients responded by mIWG criteria, with 79% progression-free at 12 months. The FDA said it does not plan an advisory committee.

Priority review and Breakthrough Therapy designation. Would be the first FDA-approved treatment for primary membranous nephropathy. In the Phase 3 MAJESTY trial, 36.9% of adults on Gazyva reached complete remission at two years versus 5.7% on tacrolimus. Genentech has said only that it expects an FDA decision by November 2026. Gazyva, a B cell-depleting antibody, is already approved for lupus nephritis and, since September 25, 2026, for idiopathic nephrotic syndrome.

An siRNA that lowers the liver's production of complement protein C5, injected under the skin every 12 weeks. The FDA granted Priority Review with a target action date in November 2026; Regeneron has not disclosed the exact day. In the Phase 3 NIMBLE trial, published in The Lancet, MG-ADL scores improved 2.3 points more than placebo at week 24. If approved, it would join Soliris, its biosimilars, Ultomiris and Zilbrysq as a C5-targeting option for AChR-positive adults.

Priority review; AstraZeneca has disclosed a Q4 window but no exact date.

Once-daily oral MC1R agonist under priority review; would be the first oral treatment for these light-sensitivity disorders. LEO Pharma has disclosed only that the action date falls by the end of February 2027.

First-in-class oral dopamine D1 receptor antagonist under priority review with Orphan Drug designation for children and adolescents with Tourette syndrome, which Teva says affects about 100,000 US children. If approved, it would be the first new Tourette treatment in more than 10 years and the first new mechanism in more than 50 years; the three approved drugs (haloperidol, pimozide, aripiprazole) all block D2 receptors and are often stopped for weight gain or movement side effects. In the 153-patient Phase 2b D1AMOND trial, ecopipam reduced tic severity on the YGTSS total tic score more than placebo at week 12; in the Phase 3 randomized withdrawal trial published in JAMA Neurology, pediatric responders kept on ecopipam had a 53% lower risk of relapse over 12 weeks than those switched to placebo. Teva has disclosed only that the action date falls late in the first quarter of 2027.

Would be the first inhaled antifibrotic for IPF. United Therapeutics has disclosed only that it expects the review to finish in late April 2027.

Next-generation enzyme replacement therapy under priority review for patients aged 2 and older with hypophosphatasia, an inherited disorder of low alkaline phosphatase activity that leaves bones and teeth poorly mineralized. The application rests on 3 Phase 3 trials (HICKORY, MULBERRY and CHESTNUT). Alexion's older enzyme therapy, Strensiq, is already approved for HPP. AstraZeneca has disclosed only that it expects an FDA decision in the first half of 2027.

Under-the-skin injection to prevent bleeding episodes in adults and children with hemophilia A, including people with inhibitors (antibodies that stop standard factor VIII replacement from working). It mimics the job of factor VIII, the clotting protein people with hemophilia A lack, and can be given once a month, every 2 weeks or weekly from a single-use prefilled pen. Novo submitted the application in September 2025 and had guided to a US decision in Q3 2026. On October 2, 2026 Novo said the FDA has extended its review because of ongoing remediation at the manufacturing facility following a pre-license inspection, and has not given a new timeline for a decision; the FDA identified no deficiencies in the clinical efficacy or safety data. Novo now aims to launch in the US in the first half of 2027, pending the decision. In the pivotal FRONTIER2 trial it significantly reduced bleeding rates compared with prior factor prophylaxis or on-demand treatment. Europe's CHMP recommended approval under the name Frehemgo on September 17, 2026.

One-time AAV gene therapy with RMAT designation; rolling BLA underway with the final module expected in Q4 2026. PTC completed its acquisition of the program from Sangamo on September 21, 2026. A PDUFA date follows once the FDA accepts the completed application.

One-time gene therapy delivered by neurosurgery into the striatum to silence the huntingtin gene; would be the first treatment shown to slow Huntington's disease. uniQure submitted the BLA for accelerated approval on September 2, 2026 and requested priority review; the FDA's filing decision is due within 60 days and no target action date has been disclosed. Holds Breakthrough Therapy, RMAT and Fast Track designations.

One-time in-office injection that makes surviving retinal cells light-sensitive, so it works regardless of which gene caused the disease. The FDA accepted the application on September 9, 2026; Nanoscope has not disclosed an exact target action date. Its October 1, 2026 announcement of priority review in Japan said it expects potential approval in both Japan and the United States in the first half of 2027.

Antibody-linked exon 44 skipping therapy designed to carry the drug into skeletal and heart muscle, for people with Duchenne whose mutations are amenable to exon 44 skipping. The FDA accepted the application for priority review under the accelerated approval pathway, based on the Phase 1/2 EXPLORE44 trial and its open-label extension; a global Phase 3 trial now underway is the confirmatory study. Novartis disclosed the acceptance on September 8, 2026 but has not disclosed a target action date.

A C5-blocking nanobody given as a once-weekly self-injection with an autoinjector, approved in Japan as Klygefa and recommended for EU approval by the CHMP at its September 14 to 17, 2026 meeting. In the 260-patient Phase 3 PREVAIL trial, MG-ADL scores improved 1.6 points more than placebo at week 26. AstraZeneca says the US application is under review but has not disclosed a target action date.

Off-the-shelf T cell therapy made from healthy donors' EBV-specific T cells, for adults and children 2 and older whose EBV+ PTLD has come back or not responded after at least one prior therapy. PTLD is an ultra-rare cancer that follows a solid organ or stem cell transplant; once first-line treatment fails, median survival is about 3 weeks after a stem cell transplant and 4 months after an organ transplant. This is the third time the application has gone to the FDA: a January 2025 complete response letter cited a third-party manufacturing site, and a January 2026 letter said the single-arm ALLELE trial could no longer support accelerated approval. Pierre Fabre resubmitted on September 30, 2026 with more patients and longer follow-up from ALLELE plus expanded access and European commercial data, after an April 2026 Type A meeting. The FDA has not yet said whether it has accepted the resubmission, and no target action date has been disclosed. Already approved in the EU, UK and Switzerland as Ebvallo.

Approved September 30, 2026, on its action date, extending Camzyos to children who weigh at least 30 kg with obstructive hypertrophic cardiomyopathy, and adding a 1 mg capsule for them. It is the first cardiac myosin inhibitor approved for anyone under 18. The approval rests on the Phase 3 SCOUT-HCM trial, in which mavacamten lowered the Valsalva outflow gradient by 48.5 mmHg at week 28 against 0.5 mmHg on placebo. The boxed warning for heart failure and the REMS program with echocardiogram monitoring carry over from the adult label. The FDA had not yet posted the revised label when this entry was written; check the drug page for label details as they are added.

Approved September 28, 2026, on its action date, to treat peripheral thyrotoxicosis (the effects of too much thyroid hormone on the body) in adults and children with MCT8 deficiency, also called Allan-Herndon-Dudley syndrome. It is the first FDA-approved treatment for the disease. Emcitate is a tablet dispersed in water and given by mouth or feeding tube, with the dose raised about every 2 weeks against blood T3, which has to be measured by LC-MS/MS because the drug makes standard T3 tests read falsely high. In the randomized withdrawal study, T3 rose in all 8 patients switched to placebo, more than in any of the 7 who stayed on the drug; it has not been shown to improve brain development. The label carries a boxed warning against use for weight loss, and the most common side effects were diarrhea, vomiting, rash and excessive sweating. Egetis expects it to be available within 8 to 10 weeks through its Egetis RareLink program and received a Rare Pediatric Disease Priority Review Voucher.

Approved September 25, 2026 as Atebrioz, 1 day ahead of its September 26th action date, to reduce the volume of total new heterotopic ossification in adults and pediatric patients 12 and older with FOP. It is a once-daily 100 mg oral ALK2 inhibitor and the third FOP treatment, after Sohonos (2023) and Pasatru (August 2026). It carries fast track, priority review, and orphan drug designations. The label warns of fetal harm based on animal studies (patients of reproductive potential must use effective contraception; pregnant patients should discontinue and contact their provider) and of interactions with certain other medications; the most common side effects are headache, joint pain, upper respiratory tract infection, nosebleeds, and nausea. Mirum told Reuters it plans to launch in October and will announce the price then.

Approved September 25, 2026 to reduce the risk of relapse in adults and children 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. Idiopathic nephrotic syndrome is the most common cause of nephrotic syndrome in children, usually starting between ages 2 and 7. In the 85-patient Phase 3 INShore trial (NCT05627557), significantly more patients on Gazyva stayed relapse-free with normal urine protein at week 52 than on mycophenolate mofetil, a common standard treatment. Gazyva was already approved for certain blood cancers and for lupus nephritis. The label carries a boxed warning for hepatitis B reactivation and progressive multifocal leukoencephalopathy, a rare brain infection; the most common side effects in this use were infections, infusion reactions and low neutrophil counts. It held Breakthrough Therapy, Orphan Drug and Priority Review designations.

Approved September 23, 2026 as Lyrfigtu, a few days ahead of its late September action date, for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 gene fusion or other rearrangement. It is the fourth FGFR drug approved for this cancer (infigratinib was later withdrawn) and the first designed to block FGFR2 more selectively than the others. The approval rests on the 116-patient REFOCUS cohort (NCT04526106): 46% of patients responded and responses lasted a median 11.8 months. The label carries warnings for eye toxicity, high blood phosphate with soft tissue mineralization, and harm to a developing baby. The FDA's notice calls it an approval rather than an accelerated approval, the pathway used for all 3 earlier FGFR drugs in this cancer.

Approved September 18, 2026, 1 day ahead of its action date, as the first treatment for ataxia in ataxia-telangiectasia, for adults and children weighing at least 15 kg (about 33 pounds). Aqneursa was already approved in 2024 for Niemann-Pick disease type C. The approval rests on a 73-patient placebo-controlled crossover study (NCT06673056) using the functional SARA ataxia scale; the most common side effects were falls, skin lacerations and urinary tract infections. IntraBio's own approval release (September 18, 2026) says the drug is commercially available now and is the first approved A-T treatment anywhere in the world.

Approved 2 days ahead of its September 19th action date as the first treatment in the history of Sanfilippo syndrome. A traditional approval on a clinical cognitive endpoint rather than the accelerated, biomarker-based approval many expected. The label covers pediatric patients with preserved neurodevelopmental function; the children in the efficacy analysis were treated at a mean age of 22 months.

Approved 19 days ahead of its September 30th action date as the first muscle-targeted treatment for SMA. The label requires patients to already be on an SMN2-targeted therapy, making it an add-on rather than a replacement.