Treatment Watch

2715 eventsUpdated today

FDA drug approvals, PDUFA decisions, orphan designations, and breakthrough therapy designations for rare diseases — sourced daily from FDA.gov.

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FDA data is sourced from FDA.gov and openFDA.
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43 events · FDA approval · 2020

Dec 21
2020
FDA approvalOrphan drug
Kalydeco (IVACAFTOR)

treatment of cystic fibrosis (CF) in patients age 4 months and older who have one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data

Acute panmyelosis with myelofibrosis· Vertex Pharmaceuticals Incorporated
Dec 21
2020
FDA approvalOrphan drug
EBANGA® (ansuvimab-zykl)

treatment of infection caused by Zaire ebolavirus in adult and pediatric patients, including neonates born to a mother who is RT-PCR positive for Zaire ebolavirus infection

Ebola hemorrhagic fever· Emergent Manufacturing Operations Baltimore LLC
Dec 18
2020
FDA approvalOrphan drug
Kineret (anakinra)

treatment of Deficiency of Interleukin-1 Receptor Antagonist (DIRA)

DITRA· Swedish Orphan Biovitrum AB (publ)
Dec 10
2020
FDA approval
ASENAPINE (ASENAPINE)FDA label ↗
· Sigmapharm Laboratories, LLC· ANDA206107
Dec 3
2020
FDA approvalOrphan drug
ORLADEYO® (berotralstat)

prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years of age and older

Peeling skin syndrome type A· BioCryst Pharmaceuticals, Inc.
Nov 25
2020
FDA approvalOrphan drug
DANYELZA® (naxitamab-gqgk)

in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy

Esthesioneuroblastoma· Y-mAbs Therapeutics, Inc.
Nov 23
2020
FDA approvalOrphan drug
Oxlumo (LUMASIRAN)FDA label ↗

treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients

Primary hyperoxaluria type 1· Alnylam Pharmaceuticals, Inc.· NDA214103
Nov 20
2020
FDA approvalOrphan drug
Zokinvy (LONAFARNIB)FDA label ↗

in patients 12 months of age and older with a body surface area (BSA) of 0.39 m2 and above (1) to reduce the risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS); and (2) for the treatment of processing-deficient Progeroid Laminopathies with either heterozygous LMNA mutation with progerin-like protein accumulation or homozygous or compound heterozygous ZMPSTE24 mutations

Hutchinson-Gilford progeria syndrome· Sentynl Therapeutics, Inc.· NDA213969
Oct 14
2020
FDA approvalOrphan drug
INMAZEB® (atoltivimab, maftivimab, and odesivimab-ebgn)

treatment of infection caused by Zaire ebolavirus in adult and pediatric patients, including neonates born to a mother who is RT-PCR positive for Zaire ebolavirus infection

Congenital enterovirus infection· Regeneron Pharmaceuticals, Inc.
Oct 13
2020
FDA approval
WAKIX (PITOLISANT HYDROCHLORIDE)FDA label ↗
· Harmony Biosciences, LLC· NDA211150
Oct 2
2020
FDA approvalOrphan drug
OPDIVO® and YERVOY® (nivolumab and ipilimumab)

YERVOY® (ipilimumab), in combination with nivolumab, is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma.

Pleural mesothelioma· Bristol-Myers Squibb Company
Sep 29
2020
FDA approvalOrphan drug
SIMPONI ARIA® (golimumab)

treatment of active polyarticular juvenile idiopathic arthritis (pJIA) in patients 2 years of age and older

Polyarticular juvenile idiopathic arthritis· Janssen Research & Development, LLC
Sep 4
2020
FDA approvalOrphan drug
GAVRETO® (pralsetinib)

treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test

MITF-related melanoma and renal cell carcinoma predisposition syndrome· Rigel Pharmaceuticals, Inc.
Sep 3
2020
FDA approvalOrphan drug
Detectnet (copper Cu 64 dotatate)

indicated for use with positron emission tomography (PET) for localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult patients

Familial gastric type 1 neuroendocrine tumor· RadioMedix Inc.
Sep 1
2020
FDA approval
ONUREG (AZACITIDINE)FDA label ↗
Juvenile myelomonocytic leukemia· Celgene Corporation· NDA214120
Aug 20
2020
FDA approvalOrphan drug
Kyprolis (carfilzomib)

Treatment of adult patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy in combination with daratumumab and dexamethasone.

Multiple myeloma· Onyx Therapeutics, Inc.
Aug 14
2020
FDA approvalOrphan drug
Enspryng (SATRALIZUMAB)FDA label ↗

treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Neuromyelitis optica spectrum disorder with anti-AQP4 antibodies· Genentech, Inc.· BLA761149
Aug 6
2020
FDA approvalOrphan drug
LAMPIT (nifurtimox)

LAMPIT is indicated in pediatric patients (birth to less than 18 years of age and weighing at least 2.5 kg) for the treatment of Chagas disease (American Trypanosomiasis), caused by Trypanosoma cruzi

American trypanosomiasis· Bayer HealthCare Pharmaceuticals, Inc.
Aug 5
2020
FDA approvalOrphan drug
BLENREP (belantamab mafodotin-blmf)

for the treatment of adults with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent (indication withdrawn)

Multiple myeloma· GlaxoSmithKline Intellectual Property Development Ltd.
Aug 5
2020
FDA approvalOrphan drug
SUPREP Bowel Prep Kit (sodium sulfate, potassium sulfate, and magnesium sulfate)

for cleansing of the colon as a preparation for colonoscopy in adult and pediatric patients 12 years of age and older

Secondary interstitial lung disease in childhood and adulthood· Braintree Laboratories, Inc.
Jul 31
2020
FDA approvalOrphan drug
MONJUVI (tafasitamab-cxix)

MONJUVI® is indicated in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT)

Diffuse large B-cell lymphoma· Incyte Corporation
Jul 24
2020
FDA approvalOrphan drug
TECARTUS® (brexucabtagene autoleucel)

TECARTUS® is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL)

Mantle cell lymphoma· Kite Pharma, Inc.
Jul 23
2020
FDA approval
IMATINIB MESYLATE (IMATINIB MESYLATE)
Myelodysplastic/myeloproliferative disease· BluePoint Laboratories· ANDA212773
Jul 21
2020
FDA approvalOrphan drug
Xywav (calcium, magnesium, potassium, and sodium oxybates)

XYWAV is indicated for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy.

Narcolepsy· Jazz Pharmaceuticals Ireland Limited
Jul 7
2020
FDA approvalOrphan drug
Inqovi (CEDAZURIDINE AND DECITABINE)FDA label ↗

Indicated for the treatment of adult patients with myelodysplastic syndromes (MDS), including previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and chronic myelomonocytic leukemia [CMML]) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

Juvenile myelomonocytic leukemia· Taiho Pharmaceutical Co., Ltd.· NDA212576
Jun 30
2020
FDA approvalOrphan drug
Dojolvi (triheptanoin)

DOJOLVI is indicated as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).

Metabolic disease due to other fatty acid oxidation disorder· Ultragenyx Pharmaceutical, Inc.
Jun 26
2020
FDA approvalOrphan drug
Lialda (mesalamine; 5-aminosalicylic acid)

Lialda (mesalamine) is indicated for the treatment of mildly to moderately active ulcerative colitis in pediatric patients weighing at least 24 kg.

Peyronie syndrome· Takeda Development Center Americas, Inc.
Jun 26
2020
FDA approvalOrphan drug
MYCAPSSA (formerly Octreolin) (octreotide (oral))

Mycapssa (octreotide) is indicated for the long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.

Fourth branchial cleft anomaly· Chiesi USA, Inc.
Jun 22
2020
FDA approvalOrphan drug
XPOVIO (selinexor)

XPOVIO is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy.

Diffuse large B-cell lymphoma· Karyopharm Therapeutics, Inc.
Jun 18
2020
FDA approvalOrphan drug
Tazverik (tazemetostat)

TAZVERIK (tazemetostat) is indicated for: the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least 2 prior systemic therapies; and the treatment of adult patients with R/R FL who have no satisfactory alternative treatment options.

Follicular lymphoma· Epizyme, Inc.
Jun 18
2020
FDA approvalOrphan drug
Crysvita (burosumab-twza)

Crysvita (burosumab-twza) is indicated for the treatment of FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized.

Oncogenic osteomalacia· Kyowa Kirin, Inc.
Jun 15
2020
FDA approval
ZEPZELCA (LURBINECTEDIN)FDA label ↗
Small cell lung cancer· Jazz Pharmaceuticals, Inc.· NDA213702
Jun 11
2020
FDA approvalOrphan drug
Uplizna (INEBILIZUMAB)FDA label ↗

Uplizna is indicated for for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

Neuromyelitis optica spectrum disorder· Horizon Therapeutics USA, Inc.· BLA761142
May 29
2020
FDA approvalOrphan drug
Tecentriq and Avastin (atezolizumab + bevacizumab)

TECENTRIQ, in combination with bevacizumab, for the treatment of patients with unresectable or metastatic hepatocellular carcinoma who have not received prior systemic therapy.

Adult hepatocellular carcinoma· Genentech, Inc.
May 14
2020
FDA approvalOrphan drug
POMALYST® (pomalidomide)

POMALYST (pomalidomide) is indicated for the treatment of adult patients with AIDS-related Kaposi sarcoma (KS) after failure of highly active antiretroviral therapy (HAART); and for the treatment of Kaposi sarcoma (KS) in adult patients who are HIV-negative.

Kaposi sarcoma· Celgene Corporation
Apr 15
2020
FDA approvalOrphan drug
Jelmyto (mitomycin)

JELMYTO® is indicated for the treatment of adult patients with low-grade Upper Tract Urothelial Cancer (LG-UTUC)

Upper tract urothelial carcinoma· UroGen Pharma, Ltd.
Apr 10
2020
FDA approvalOrphan drug
Koselugo (selumetinib)

Pediatric patients 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas. Initially approved April 10, 2020. CONTEXT: selumetinib was initially approved only for pediatric patients — adult NF1-PN patients had no approved treatment until mirdametinib (Gomekli) in February 2025.

Neurofibromatosis type 1· AstraZeneca / Alexion· NDA 213756
Mar 6
2020
FDA approvalOrphan drug
Isturisa (osilodrostat)

ISTURISA is a cortisol synthesis inhibitor indicated for the treatment of adult patients with Cushing®s disease for whom pituitary surgery is not an option or has not been curative.

Cushing disease· Recordati Rare Diseases, Inc
Mar 2
2020
FDA approvalOrphan drug
Sarclisa (isatuximab-irfc)

SARCLISA is indicated, in combination with pomalidomide and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor.

Multiple myeloma· Sanofi-Aventis U.S. LLC
Feb 27
2020
FDA approval
NURTEC ODT (RIMEGEPANT SULFATE)FDA label ↗
Nodular regenerative hyperplasia of the liver· Pfizer Laboratories Div Pfizer Inc· NDA212728
Jan 24
2020
FDA approvalOrphan drug
Dificid (fidaxomicin)

DIFICID® is indicated in adult and pediatric patients aged 6 months and older for the treatment of C. difficile-associated diarrhea (CDAD).

Congenital enterovirus infection· Cubist Pharmaceuticals LLC, a subsidiary of Merck Sharp & Dohme LLC
Jan 10
2020
FDA approvalOrphan drug
VALTOCO (diazepam (intranasal))

Acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient®s usual seizure pattern in patients with epilepsy 6 years of age and older.

Folinic acid-responsive seizures· Neurelis Pharmaceuticals, Inc.
Jan 9
2020
FDA approvalOrphan drug
Ayvakit (avapritinib)

Treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations

Gastrointestinal stromal tumor· Blueprint Medicines Corporation

Not medical advice — always consult your healthcare provider before making treatment decisions.

Data sourced from FDA.gov and openFDA, updated daily. Dates are subject to change. Always verify with FDA.gov for official decisions.