Orphan drug approvals FDA EMA 2014

Orphan Drug Approvals in 2014

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Generic Name: trametinib and dabrafenib

Trade Name: Mekinist And Tafinlar

Indication: Treatment of Stage IIb through IV melanoma

Marketing Approval Date: 01-08-2014/01-09-2014

Exclusivity End Date: 01-08-2021/01-09-2021

Sponsor: GlaxoSmithKline

Generic Name: tasimelteon

Trade Name: Hetlioz

Indication: Non-24-hour sleepwake disorder in blind individuals without light perception

Marketing Approval Date: 01-31-2014

Exclusivity End Date: 01-31-20121

Sponsor: Vanda Pharmaceuticals

Generic Name: ibrutinib

Trade Name: Imbruvica

Indication: Treatment of chronic lymphocytic leukemia (CLL)

Marketing Approval Date: 02-12-2014

Exclusivity End Date: 02-12-2021

Sponsor: Pharmacyclics

Generic Name: elosulfase alfa

Trade Name: Vimizim

Indication: Use in the treatment of mucopolysaccharidosis (MPS) Type IV A (Morquio A syndrome)

Marketing Approval Date: 02-14-2014

Exclusivity End Date: 02-14-2021

Sponsor: BioMarin

Generic Name: droxidopa

Trade Name: Northera

Indication: Treatment of neurogenic symptomatic orthostatic hypotension in patients with primary autonomic failure, dopamine-beta-hydroxylase deficiency, and nondiabetic autonomic neuropathy.

Marketing Approval Date: 02-18-2014

Exclusivity End Date: 02-18-2021

Sponsor: Lundbeck

Generic Name: metreleptin

Trade Name: Myalept

Indication: Treatment of metabolic disorders secondary to lipodystrophy

Marketing Approval Date: 02-24-2014

Exclusivity End Date: N/A

Sponsor: Amylin Pharmaceuticals

Generic Name: propranolol

Trade Name: Hemangeol

Indication: Treatment of proliferating infantile hemangiomas requiring systemic therapy

Marketing Approval Date: 03-14-2014

Exclusivity End Date: N/A

Sponsor: Pierre Fabre Dermatologie

Generic Name: Impavido

Trade Name: miltefosine

Indication: Treatment of leishmaniasis.

Marketing Approval Date: 03-19-2014

Exclusivity End Date: 03-19-2021

Sponsor: Knight Therapeutics

Generic Name: Coagulation factor IX (recombinant), Fc fusion protein

Trade Name: Alprolix

Indication: Control and prevention of hemorrhagic episodes in patients with hemophilia B (congenital factor IX deficiency or Christmas disease)

Marketing Approval Date: 03-28-2014

Exclusivity End Date: 03-28-2021

Sponsor: Biogen Idec

Generic Name: ecallantide

Trade Name: Kalbitor

Indication: Treatment of acute attacks of hereditary angioedema (HAE) in patients 12 years of age and older

Marketing Approval Date: 03-28-2014

Exclusivity End Date: 03-28-2021

Sponsor: Dyax Corp.

Generic Name: ethiodized oil injection

Trade Name: Lipiodol

Indication: Selective hepatic intra-arterial use for imaging tumors in adults with known hepatocellular carcinoma

Marketing Approval Date: 04-04-2014

Exclusivity End Date: 04-04-2021

Sponsor: Guerbet LLC

Generic Name: ofatumumab

Trade Name: Arzerra

Indication: Ofatumumab in combination with chlorambucil, for the treatment of previously untreated patients with chronic lymphocytic leukemia (CLL) for whom fludarabine-based therapy is considered inappropriate.

Marketing Approval Date: 04-17-2014

Exclusivity End Date: 04-17-2021

Sponsor: Glaxo Group Limited d/b/a GlaxoSmithKline

Generic Name: ramucirumab

Trade Name: Cyramza

Indication: Treatment of advanced gastric or gastro-esophageal junction adenocarcinoma, as a single agent or in combination with paclitaxel, after prior fluoropyrimidine- or platinium-containing chemotherapy.

Marketing Approval Date: 11-05-2014

Exclusivity End Date: N/A

Sponsor: Eli Lilly and Company

Generic Name: siltuximab

Trade Name: Sylvant

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

Marketing Approval Date: 04-23-2014

Exclusivity End Date: 04-23-2021

Sponsor: Janssen Biotech

Generic Name: mercaptopurine oral solution

Trade Name: Purixan

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

Marketing Approval Date: 04-28-2014

Exclusivity End Date: 04-28-2021

Sponsor: Nova Laboratories Limited

Generic Name: ceritinib

Trade Name: Zykadia

Indication: Treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib.

Marketing Approval Date: 04-29-2014

Exclusivity End Date: 04-29-2021

Sponsor: Novartis Pharmaceuticals Corp.

Generic Name: antihemophilic factor (recombinant), Fc fusion protein

Trade Name: Eloctate

Indication: Treatment of adults and children with Hemophilia A (congenital Factor VIII deficiency) for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

Marketing Approval Date: 06-06-2014

Exclusivity End Date: 06-06-2021

Sponsor: Biogen Idec Inc.

Generic Name: technetium Tc 99m tilmanocept

Trade Name: Lymphoseek

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

Marketing Approval Date: 06-13-2014

Exclusivity End Date: 06-13-2021

Sponsor: Navidea Biopharmaceuticals

Generic Name: coagulation factor VIIa (recombinant)

Trade Name: Novoseven Rt

Indication: Treatment of bleeding episodes and peri-operative management in adults and children with Glanzmann’s thrombasthenia with refractoriness to platelet transfusions, with or without antibodies to platelets.

Marketing Approval Date: 07-02-2014

Exclusivity End Date: 07-02-2021

Sponsor: Novo Nordisk Inc.

Generic Name: Belinostat

Trade Name: Beleodaq

Indication: Treatment of peripheral T-cell lymphoma (PTCL).

Marketing Approval Date: 07-03-2014

Exclusivity End Date: 07-03-2021

Sponsor: Spectrum Pharmaceuticals, Inc.

Generic Name: C1-esterase inhibitor (recombinant)

Trade Name: Ruconest

Indication: Treatment of acute attacks of hereditary angioedema (HAE) in adult and adolescent patients.

Marketing Approval Date: 07-16-2014

Exclusivity End Date: 07-16-2021

Sponsor: Santarus, Inc.

Generic Name: dantrolene sodium suspension for injection

Trade Name: Ryanodex

Indication: Treatment of malignant hyperthermia in conjunctin with appropriate supportive measures.

Marketing Approval Date: 07-22-2014

Exclusivity End Date: N/A

Sponsor: Eagle Pharmaceuticals, Inc.

Generic Name: idelalisib

Trade Name: Zydelig

Indication: (1) Relapsed chronic lymphocytic leukemia (CLL), in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy due to other co-morbidities; and (2) Relapsed small lymphocytic lymphoma (SLL) in patients who have received at least two prior systemic therapies.

Marketing Approval Date: 07-23-2014

Exclusivity End Date: 07-23-2021

Sponsor: Gilead Sciences, Inc

Generic Name: ibrutinib

Trade Name: Imbruvica

Indication: Treatment of chronic lymphocytic leukemia with 17p deletion

Marketing Approval Date: 07-28-2014

Exclusivity End Date: 07-28-2021

Sponsor: Pharmacyclics, Inc.

Generic Name: Recombinant human acid alpha-glucosidase; alglucosidase alfa

Trade Name: 1. Myozyme 2. Lumizyme

Indication: (Lumizyme) Patients with Pompe disease (acid alpha-glucosidase (GAA deficiency)).

Marketing Approval Date: 08-01-2014

Exclusivity End Date: 08-01-2021

Sponsor: Genzyme Corporation

Generic Name: eliglustat

Trade Name: Cerdelga

Indication: 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.

Marketing Approval Date: 08-19-2014

Exclusivity End Date: 08-19-2021

Sponsor: Genzyme Corporation

Generic Name: eltrombopag

Trade Name: Promacta

Indication: Treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy.

Marketing Approval Date: 08-26-2014

Exclusivity End Date: 08-26-2021

Sponsor: GlaxoSmithKline LLC

Generic Name: pembrolizumab

Trade Name: Keytruda

Indication: Treatment of patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor.

Marketing Approval Date:

Exclusivity End Date:

Sponsor: Merck, Sharp & Dohme Corp.

Generic Name: adalimumab

Trade Name: Humira

Indication: 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. (Orphan approval for patients from 6 through 16 years of age.)

Marketing Approval Date: 09-23-2014

Exclusivity End Date: 09-23-2021

Sponsor: AbbVie Inc.

Generic Name: adalimumab

Trade Name: Humira

Indication: Reducing signs and symptoms of moderately to severely active polyarticular juvenile idiopathic arthritis in patients 2 years of age and older.

Marketing Approval Date: 09-30-2014

Exclusivity End Date: 09-30-2021

Sponsor: AbbVie Inc.

Generic Name: bortezomib

Trade Name: Velcade For Injection

Indication: Treatment of patients with mantle cell lymphoma

Marketing Approval Date: 10-08-2014

Exclusivity End Date: N/A

Sponsor: Millennium Pharmaceuticals, Inc.

Generic Name: nintedanib

Trade Name: Ofev

Indication: Treatment of idiopathic pulmonary fibrosis

Marketing Approval Date: 10-15-2014

Exclusivity End Date: 10-15-2021

Sponsor: Boehringer Ingelheim Pharmaceuticals, Inc.

Generic Name: pirfenidone

Trade Name: Esbriet

Indication: Treatment of idiopathic pulmonary fibrosis

Marketing Approval Date: 10-15-2014

Exclusivity End Date: 10-15-2021

Sponsor: Intermune

Generic Name: antihemophilic factor (recombinant), porcine sequence

Trade Name: Obizur

Indication: Treatment of bleeding episodes in adults with acquired hemophilia A.

Marketing Approval Date: 10-23-2014

Exclusivity End Date: 10-23-2021

Sponsor: Baxter Healthcare Corporation

Generic Name: ramucirumab

Trade Name: Cyramza

Indication: Treatment of advanced gastric or gastro-esophageal junction adenocarcinoma, as a single agent or in combination with paclitaxel, after prior fluoropyrimidine- or platinium-containing chemotherapy.

Marketing Approval Date: 11-05-2014

Exclusivity End Date: 11-05-2021

Sponsor: Eli Lilly and Company

Generic Name: bevacizumab

Trade Name: Avastin

Indication: In combination with paclitaxel, pegylated liposomal doxorubicin, or topotecan for treatment of patients with platinum-resistant, recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who received no more than 2 prior chemotherapy regimens

Marketing Approval Date: 11-14-2014

Exclusivity End Date: 11-14-2021

Sponsor: Genentech, Inc

Generic Name: cinacalcet

Trade Name: Sensipar

Indication: Treatment of hypercalcemia in adult patients with primary hyperparathyroidism for whom parathyroidectomy would be indicated on the basis of serum calcium levels, but who are unable to undergo parathyroidectomy.

Marketing Approval Date: 11-21-2014

Exclusivity End Date: 11-21-2021

Sponsor: Amgen, Inc.

Generic Name: blinatumomab

Trade Name: N/A

Indication: Treatment of Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).

Marketing Approval Date: 12-03-2014

Exclusivity End Date: 12-03-2021

Sponsor: Amgen, Inc.

Generic Name: ruxolitinib

Trade Name: Jakafi

Indication: Treatment of patients with polycythemia vera who have had an inadequate response to or are intolerant of hydroxyurea.

Marketing Approval Date: 12-04-2014

Exclusivity End Date: 12-04-2021

Sponsor: Incyte Corporation

Generic Name: denosumab

Trade Name: Xgeva

Indication: To treat hypercalcemia of malignancy refractory to biphosphonate therapy.

Marketing Approval Date: 12-05-2014

Exclusivity End Date: 12-05-2021

Sponsor: Amgen, Inc.

Generic Name: nivolumab

Trade Name: Opdivo

Indication: treatment for patients with unresectable or metastatic (advanced) melanoma who no longer respond to other drugs.

Marketing Approval Date: 12-22-2014

Exclusivity End Date: N/A

Sponsor: Bristol-Myers Squibb

Generic Name: bedaquiline

Trade Name: Sirturo

Indication: Indicated for use as part of an appropriate combination regimen for pulmonary multidrug resistant tuberculosis (MDR TB) in adult patients when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability. See sections 4.2, 4.4 and 5.1.
Consideration should be given to official guidance on the appropriate use of antibacterial agents.

Marketing Approval Date: 03-05-2014

Conditional Approval: YES

Sponsor: Janssen-Cilag International N.V.

Generic Name: cabozantinib

Trade Name: Cometriq

Indication: Treatment of adult patients with progressive, unresectable locally advanced or metastatic medullary thyroid carcinoma

Marketing Approval Date: 03-21-2014

Conditional Approval: YES

Sponsor: TMC Pharma Services Ltd

Generic Name: riociguat

Trade Name: Adempas

Indication: Chronic thromboembolic pulmonary hypertension (CTEPH) Adempas is indicated for the treatment of adult patients with WHO Functional Class (FC) II to III with
inoperable CTEPH,
persistent or recurrent CTEPH after surgical treatment,
to improve exercise capacity (see section 5.1). Pulmonary arterial hypertension (PAH) Adempas, as monotherapy or in combination with endothelin receptor antagonists, is indicated for the treatment of adult patients with pulmonary arterial hypertension (PAH) with WHO Functional Class (FC) II to III to improve exercise capacity. Efficacy has been shown in a PAH population including aetiologies of idiopathic or heritable PAH or PAH associated with connective tissue disease (see section 5.1).

Marketing Approval Date: 03-27-2014

Conditional Approval: NO

Sponsor: Bayer Pharma AG

Generic Name: cholic acid

Trade Name: Kolbam (previously Cholic Acid FGK)

Indication: Kolbamis indicated for the treatment of inborn errors in primary bile acid synthesis due to Sterol 27-hydroxylase (presenting as cerebrotendinous xanthomatosis, CTX) deficiency, 2- (or a-) methylacyl-CoA racemase (AMACR) deficiency or Cholesterol 7a-hydroxylase (CYP7A1) deficiency in infants, children and adolescents aged 1 month to 18 years and adults.

Marketing Approval Date: 04-04-2014

Conditional Approval: NO

Sponsor: FGK Representative Service GmbH

Generic Name: para-aminosalicylic acid

Trade Name: Granupas (previously Para-aminosalicylic acid Lucane)

Indication: Granupas is indicated for use as part of an appropriate combination regimen for multi-drug resistant tuberculosis in adults and paediatric patients from 28 days of age and older when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability (see section 4.4).
Consideration should be given to official guidance on the appropriate use of antibacterial agents.

Marketing Approval Date: 04-07-2014

Conditional Approval: NO

Sponsor: Lucane Pharma

Generic Name: elosulfase alfa

Trade Name: Vimizim

Indication: Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages.

Marketing Approval Date: 04-28-2014

Conditional Approval: NO

Sponsor: BioMarin Europe Ltd

Generic Name: delamanid

Trade Name: Deltyba

Indication: Deltyba is indicated for use as part of an appropriate combination regimen for pulmonary multi-drug resistant tuberculosis (MDR-TB) in adult patients when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability.
Consideration should be given to official guidance on the appropriate use of antibacterial agents.

Marketing Approval Date: 04-28-2014

Conditional Approval: YES

Sponsor: Otsuka Novel Products GmbH

Generic Name: siltuximab

Trade Name: Sylvant

Indication: Sylvant is indicated for the treatment of adult patients with multicentric Castlemans disease (MCD who are human immunodeficiency virus (HIV) negative and human herpesvirus-8 (HHV-8) negative.

Marketing Approval Date: 05-22-2014

Conditional Approval: NO

Sponsor: Janssen-Cilag International NV

Generic Name: obinutuzumab

Trade Name: Gazyvaro

Indication: Gazyvaro in combination with chlorambucil is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL) and with comorbidities making them unsuitable for full-dose fludarabine based therapy

Marketing Approval Date: 07-23-2014

Conditional Approval: NO

Sponsor: Roche Registration Ltd

Generic Name: ataluren

Trade Name: Translarna

Indication: Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older.

Marketing Approval Date: 07-31-2014

Conditional Approval: YES

Sponsor: PTC Therapeutics International Limited

Generic Name: ibrutinib

Trade Name: Imbruvica

Indication: Imbruvica is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL).Imbruvicais indicated for the treatment of adult patients with chronic lymphocytic leukaemia (CLL) who have received at least one prior therapy, or in first line in the presence of 17p deletion or TP53 mutation in patients unsuitable for chemo immunotherapy.

Marketing Approval Date: 10-21-2014

Conditional Approval: NO

Sponsor: Janssen-Cilag International NV

Generic Name: olaparib

Trade Name: Lynparza

Indication: Lynparza is indicated as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed BRCA-mutated (germline and/or somatic) high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete response or partial response) to platinum-based chemotherapy.

Marketing Approval Date: 12-16-2014

Conditional Approval: NO

Sponsor: AstraZeneca AB

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