Treatment Watch

2715 eventsUpdated today

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

FDA data is sourced from FDA.gov and openFDA.
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24 events · FDA approval · 2015

Dec 22
2015
FDA approvalOrphan drug
Uptravi (selexipag)

Indicated for PAH to delay disease progression and reduce hospitalization risk.

Pulmonary arterial hypertension· Janssen
Dec 7
2015
FDA approvalOrphan drug
Bendeka (bendamustine for 50 ml admixture)

Treatment of patients with chronic lymphocytic leukemia (CLL). Efficacy relative to first line therapies other than chlorambucil has not been established.

T-cell prolymphocytic leukemia· Eagle Pharmaceuticals, Inc.
Nov 20
2015
FDA approvalOrphan drug
Ninlaro (ixazomib citrate)

Use in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy

Multiple myeloma· Takeda Development Center Americas, Inc.
Nov 18
2015
FDA approval
NARCAN (NALOXONE HYDROCHLORIDE NASAL)FDA label ↗
· Emergent Devices Inc.· NDA208411
Nov 13
2015
FDA approvalOrphan drug
Tagrisso (osimertinib)

For treatment of patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC), as detected by an FDA-approved test, who have progressed on or after EGFR TKI therapy

Non-small cell lung cancer· AstraZeneca Pharmaceuticals LP
Nov 13
2015
FDA approvalOrphan drug
Neulasta (Pegfilgrastim)

To increase survival in patients acutely exposed to myelosuppressive doses of radiation (Hematopoietic Subsyndrome of Acute Radiation Syndrome)

Difference of sex development of gynecological interest· Amgen, Inc.
Oct 28
2015
FDA approvalOrphan drug
Yervoy (ipilimumab)

For the adjuvant treatment of patients with cutaneous melanoma with pathologic involvement of regional lymph nodes of more than 1 mm, who have undergone complete resection including total lymphadenectomy.

Rare parathyroid disease and phosphocalcic metabolism anomaly· Bristol-Myers Squibb Company
Oct 27
2015
FDA approvalOrphan drug
IMLYGIC (talimogene laherparepvec)

Indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery

Melanoma· BioVex, Inc. (subsidiary of Amgen)
Oct 23
2015
FDA approvalOrphan drug
Strensiq (ASFOTASE ALFA)FDA label ↗

For the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP)

Prenatal benign hypophosphatasia· Alexion Pharmaceuticals, Inc.· BLA125513
Oct 23
2015
FDA approvalOrphan drug
Yondelis (trabectedin)

For the treatment of patients with unresectable or metastatic liposarcoma or leiomyosarcoma who received a prior anthracycline-containing regimen

Alveolar soft tissue sarcoma· Janssen Research & Development, LLC
Sep 9
2015
FDA approvalOrphan drug
HUMIRA (adalimumab)

Treatment of moderate to severe hidradenitis suppurativa

Hidradenitis suppurativa· AbbVie, Inc.
Aug 27
2015
FDA approvalOrphan drug
Repatha (evolocumab)

As an adjunct to diet and other LDL-lowering therapies (e.g., statins, ezetimibe, LDL apheresis) in patients with HoFH who require additional lowering of LDL-C.

Homozygous familial hypercholesterolemia· Amgen Inc.
Aug 20
2015
FDA approval
DEXMEDETOMIDINE HYDROCHLORIDE (DEXMEDETOMIDINE HYDROCHLORIDE)
Combined hyperlipidemia· Sun Pharmaceuticals Industries, Inc.· ANDA202126
Aug 19
2015
FDA approval
IBANDRONATE SODIUM (IBANDRONATE SODIUM)FDA label ↗
· Eugia US LLC· ANDA205332
Jul 13
2015
FDA approvalOrphan drug
Iressa (gefitinib)

For the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) substitution mutations as detected by an FDA-approved test

Non-small cell lung cancer· AstraZeneca Pharmaceuticals LP
Jul 2
2015
FDA approval
ORKAMBI (LUMACAFTOR AND IVACAFTOR)FDA label ↗
Cystic fibrosis· Vertex Pharmaceuticals Incorporated· NDA206038
May 6
2015
FDA approvalOrphan drug
Anavip (crotalidae immune F(ab')2 (equine))

Management of adult and pediatric patients with North American rattlesnake envenomation

Envenomization by Bothrops lanceolatus· Rare Disease Therapeutics, Inc.
Mar 30
2015
FDA approvalOrphan drug
Neupogen (filgrastim)

To increase survival in patients acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome).

Difference of sex development of gynecological interest· Amgen, Inc.
Mar 17
2015
FDA approvalOrphan drug
Cholbam (CHOLIC ACID)FDA label ↗

Treatment of bile acid synthesis disorders due to single enzyme defects

Rare inborn errors of metabolism· Mirum Pharmaceuticals Inc.· NDA205750
Mar 10
2015
FDA approvalOrphan drug
Unituxin (dinutuximab)

For use in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2) and 13-cis-retinoic acid (RA), for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy

Esthesioneuroblastoma· United Therapeutics Corporation
Feb 23
2015
FDA approvalOrphan drug
Farydak (panobinostat)

FARYDAK (panobinostat) in combination with bortezomib (BTZ) and dexamethasone (DEX) for the treatment of patients with multiple myeloma who have received at least 2 prior regimens, including bortezomib and an immunomodulatory agent

Multiple myeloma· zr pharma& GmbH
Jan 30
2015
FDA approval
SCOPOLAMINE TRANSDERMAL SYSTEM (SCOPOLAMINE TRANSDERMAL SYSTEM)FDA label ↗
· Padagis Israel Pharmaceuticals Ltd· ANDA078830
Jan 23
2015
FDA approvalOrphan drug
Natpara (parathyroid hormone)

An adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism

Rare hypoparathyroidism· Takeda Pharmaceuticals U.S.A., Inc.
Jan 9
2015
FDA approval
DUOPA (CARBIDOPA AND LEVODOPA)FDA label ↗
Prader-Willi-like syndrome· AbbVie Inc.· NDA203952

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.