Treatment Watch

2591 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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21 events · FDA approval · 2014

Dec 12
2014
FDA approvalOrphan drug
Abilify (aripiprazole)

Treatment of pediatric patients with Tourette's

Tourette syndrome· Otsuka Pharmaceutical Development
Oct 23
2014
FDA approvalOrphan drug
OBIZUR (antihemophilic factor (recombinant), porcine sequence)

Treatment of bleeding episodes in adults with acquired hemophilia A.

Rare thrombotic disorder due to a coagulation factors defect· Takeda Development Center Americas, Inc.
Oct 15
2014
FDA approvalOrphan drug
Ofev (NINTEDANIB)FDA label ↗

Treatment of idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis· Boehringer Ingelheim Pharmaceuticals, Inc.· NDA205832
Oct 15
2014
FDA approvalOrphan drug
Pirfenidone (PIRFENIDONE)

Treatment of idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis· AvKARE· ANDA212569
Sep 23
2014
FDA approvalOrphan drug
HUMIRA (adalimumab)

Reducing signs and symptoms and inducing and maintaining clinical remission in patients 6 years of age and older with moderately to severely active Crohn's disease who have had an inadequate response to corticosteroids or immunomodulators such as azathioprine, 6-mercaptopurine, or methotrexate.

Pediatric-onset Graves disease· AbbVie, Inc.
Aug 19
2014
FDA approvalOrphan drug
Cerdelga (ELIGLUSTAT)

Long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.

Fetal Gaucher disease· Genzyme Corporation
Jul 23
2014
FDA approvalOrphan drug
Zydelig (idelalisib)

Zydelig is indicated for the treatment of patients with relapsed follicular B-cell non-Hodgkin lymphoma (FL) who have received at least two prior systemic therapies.

Follicular lymphoma· Gilead Sciences, Inc.
Jul 22
2014
FDA approvalOrphan drug
RYANODEX (dantrolene sodium suspension for injection)

Treatment of malignant hyperthermia in conjunction with appropriate supportive measures and for the prevention of malignant hyperthermia in patients at high risk.

Multiple pterygium-malignant hyperthermia syndrome· Eagle Pharmaceuticals, Inc.
Jul 3
2014
FDA approvalOrphan drug
Beleodaq (Belinostat)

Treatment of patients with relapsed or refractory peripheral T-cell lymphoma

Primary cutaneous peripheral T-cell lymphoma not otherwise specified· Acrotech Biopharma LLC
Jun 13
2014
FDA approvalOrphan drug
LYMPHOSEEK (technetium Tc 99m tilmanocept)

Guiding sentinel lymph node biopsy, using a hand-held gamma counter in patients with clinically node negative squamous cell carcinoma of the oral cavity.

Immunodeficiency with isotype or light chain deficiencies with normal number of B-cells· Cardinal Health 414, LLC
Jun 9
2014
FDA approval
DULOXETINE HYDROCHLORIDE (DULOXETINE)
· Alembic Pharmaceuticals Limited· ANDA202949
Jun 6
2014
FDA approvalOrphan drug
Eloctate (antihemophilic factor (recombinant), Fc fusion protein)

Indicated in adults and children with Hemophilia A (congenital Factor VIII deficiency) for on-demand treatment and control of bleeding episodes, perioperative management of bleeding, and routine prophylaxis to reduce the frequency of bleeding episodes.

Moderate hemophilia A· Bioverativ Therapeutics, Inc.
Apr 28
2014
FDA approvalOrphan drug
Purixan (mercaptopurine oral solution)

Treatment of patients with acute lymphoblastic leukemia as part of a combination regimen.

Acute myeloblastic leukemia with maturation· Nova Laboratories Limited
Apr 23
2014
FDA approvalOrphan drug
SYLVANT (siltuximab)

Treatment of patients with multicentric Castleman's disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpesvirus-8 (HHV-8) negative.

Unicentric Castleman disease· Recordati Rare Diseases Inc.
Apr 4
2014
FDA approvalOrphan drug
Lipiodol (ethiodized oil injection)

Selective hepatic intra-arterial use for imaging tumors in adults with known hepatocellular carcinoma (HCC)

Pediatric hepatocellular carcinoma· Guerbet LLC
Mar 28
2014
FDA approvalOrphan drug
ALPROLIX (Coagulation factor IX (recombinant), Fc fusion protein)

Adults and children with Hemophilia B for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

Multisystem inflammatory syndrome in children and adults· Bioverativ Therapeutics, Inc.
Mar 19
2014
FDA approvalOrphan drug
IMPAVIDO (miltefosine)

Treatment of visceral leishmaniasis due to Leishmania donovani; cutaneous leishmaniasis due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis; and mucosal leishmaniasis due to Leishmania braziliensis.

Leishmaniasis· Knight Therapeutics (USA)
Mar 11
2014
FDA approval
TOPIRAMATE (TOPIRAMATE)FDA label ↗
Lennox-Gastaut syndrome· Upsher-Smith Laboratories, LLC· NDA205122
Feb 24
2014
FDA approvalOrphan drug
MYALEPT (metreleptin)

Adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.

AKT2-related familial partial lipodystrophy· Chiesi Farmaceutici S.p.A.
Feb 18
2014
FDA approvalOrphan drug
NORTHERA (droxidopa)

Treatment of orthostatic dizziness, lightheadedness, or the "feeling that you are about to black out" in adult patients with symptomatic neurogenic orthostatic hypotension caused by primary autonomic failure (Parkinson's disease, multiple system atrophy, and pure autonomic failure), dopamine beta-hydroxylase deficiency, and non-diabetic autonomic neuropathy.

PrP systemic amyloidosis· Lundbeck LLC
Feb 7
2014
FDA approval
AMMONIA N 13 (NH3N13)
· Washington University School of Medicine· ANDA204506

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.