Company
(location)

Product

Description

Indication

Status

Date

CANCER

Biolinerx Ltd. (Jerusalem)

BL-8040

A short peptide that functions as a high-affinity antagonist for chemokine receptor CXCR4

Acute myeloid leukemia

FDA confirmed orphan drug designation for BL-8040, in combination with granulocyte colony-stimulating factor, as a treatment for stem cell mobilization prior to an autologous or allogeneic transplantation

1/24/14

Cell Therapeutics Inc. (Seattle)

IND 075503

Tosedostat; a first-in-class selective inhibitor of aminopeptidases

Blood-related cancers

Received word from the FDA that the partial clinical hold has been removed and all studies under way may continue

1/3/14

CARDIOVASCULAR

Chelsea Therapeutics International Ltd. (Charlotte, N.C.)

Northera

Droxidopa

Symptomatic neurogenic orthostatic hypotension

FDA committee voted 16-1 to recommend the drug's approval in patients with primary autonomic failure, dopamine beta hydroxylase deficiency and nondiabetic autonomic neuropathy

1/15/14

CENTRAL NERVOUS SYSTEM

Avanir Pharmaceuticals Inc. (Aliso Viejo, Calif.)

AVP-825

Uses Optinose AS' closed-palate breath powered intranasal delivery system

Migraine

Submitted an NDA to the FDA for AVP-825 for the acute treatment of migraine

1/31/14

Orexigen Therapeutics Inc. (San Diego)

Contrave

Naltrexone sustained release/bupropion

Obesity

FDA considered the recent resubmission of the NDA to be a complete response to the Jan. 31, 2011, action letter

1/8/14

Salix Pharmaceuticals Ltd. (Raleigh, N.C.)

Budesonide

2-mg rectal foam

Active mild to moderate distal ulcerative colitis

FDA accepted its NDA

1/31/14

Iroko Pharmaceuticals LLC (Philadelphia)

Zorvolex

Diclofenac, a lower-dose nonsteroidal anti-inflammatory drug

Osteoarthritis pain in adults

FDA accepted for review the sNDA

1/16/14

Aeolus Pharmaceuticals Inc. (Mission Viejo, Calif.)

AEOL 10150

Antioxidant drug

Acute radiation syndrome

FDA granted orphan status for use in patients exposed to radiation following a nuclear accident or detonation in order to treat or mitigate acute radiation syndrome

1/27/14

MISCELLANEOUS

Aeterna Zentaris Inc. (Quebec City)

Macimorelin acetate

Ghrelin agonist

Adult growth hormone deficiency

FDA has accepted for filing the company's NDA

1/7/14

Alexion Pharmaceuticals Inc. (Cheshire, Conn.)

Soliris

Eculizumab

To prevent delayed graft function in renal transplant patients

FDA granted orphan drug designation

1/22/14

Amag Pharmaceuticals Inc. (Waltham, Mass.)

Feraheme

Ferumoxytol injection

Iron deficiency anemia

FDA confirmed that it is reviewing the sNDA for Feraheme in adult patients who failed or could not tolerate oral iron; FDA issued a complete response letter

1/23/14

Brabant Pharma (London)

Brabafen

Low-dose fenfluramine

Dravet syndrome

FDA and European Commission granted orphan drug designation to treat children with Dravet syndrome

1/10/14

Clarus Therapeutics Inc. (Northbrook, Ill.)

Rextoro

Oral testosterone replacement product

Testosterone deficiency

Submitted an NDA to the FDA

1/15/14

NPS Pharmaceuticals Inc. (Bedminster, N.J.)

Natpara

Recombinant human parathyroid hormone 1-84

Hypopara-thyroidism

FDA has accepted and filed for review the company’s BLA

1/9/14

Regenerx Biopharma-ceuticals Inc. (Rockville, Md.)

Tbeta4

Thymosin beta 4

Neurotrophic keratopathy

Received orphan drug designation from the FDA

1/6/14

Vivus Inc. (Mountain View, Calif.) and Auxilium Pharmaceuticals Inc. (Chesterbrook, Pa.)

Stendra

Avanafil

Erectile dysfunction

FDA accepted the sNDA proposing to revise prescribing information for Stendra with efficacy and safety information from Study TA-501, a multicenter, placebo-controlled study designed to assess the efficacy of two dosage strengths of Stendra approximately 15 minutes after dosing

1/22/14

RESPIRATORY

Alitair Pharmaceuticals Inc. (Morristown, N.J.)

Erdotin

Erdosteine

Chronic obstructive pulmonary disease and chronic bronchitis

FDA granted orphan drug designation

1/8/14


Notes

BLA = Biologics license application; CMA = Continuous marketing application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; PDUFA = Prescription Drug User Fee Act; SPA = Special protocol assessment.

Public biotech company stock symbols can be found in the stock report located on the last two pages of this issue.

The date indicated refers to the BioWorld Today issue in which the news item can be found.