FDA Submissions, Approvals And Other Actions: November 2004

Company*
(Country;
Symbol)

Product

Description

Indication

Status (Date)

AUTOIMMUNE

Biogen Idec
Inc. (BIIB) and
Elan Corp. plc
(Ireland)

Tysabri (formerly Antegren)

Natalizumab; humanized monoclonal antibody; alpha-4 antagonist in the selective adhesion molecule inhibitor class

Multiple sclerosis

The FDA granted accelerated approval to the drug for treating relapsing forms of MS to reduce the frequency of clinical relapses (11/23)

CANCER

Genzyme
Corp. (GENZ)

Thyrogen (FDA-approved)

Thyrotropin alfa for injection; a recombinant human thyroid-stimulating hormone

Ablation of remnant thyroid tissue

Submitted an NDA supplement to the FDA seeking the additional indication; the product already is approved as a diagnostic tool (11/3)

Marshall
Edwards Inc.
(MSHL)

Phenoxodiol

Agent designed to induce apoptosis by removing intracellular proteins XIAP and c-FLIP

Ovarian cancer

The FDA granted fast-track status to the product for treating recurrent ovarian cancer (11/4)

Millennium
Pharmaceuticals
Inc. (MLNM)

Velcade (FDA-approved)

Bortezomib; proteasome inhibitor

Mantle-cell lymphoma

The FDA granted the product fast- track status in treating relapsed and refractory MCL (11/2)

OSI
Pharmaceuticals
Inc. (OSIP) and
Genentech
Inc. (NYSE:DNA)

Tarceva

Erlotinib HCl; small-molecule HER1/EGFR inhibitor

Advanced non- small-cell lung cancer

The FDA approved the drug for treating patients with locally advanced or metastatic NSCLC after failure of at least one prior chemotherapy regimen (11/18)

SuperGen
Inc. (SUPG)

Orathecin

Rubitecan; topoisomerase- I inhibitor in capsule formulation

Pancreatic cancer

The FDA extended its review period 90 days, to Feb. 26, 2005, after additional data were submitted (11/29)

SuperGen
Inc. (SUPG)

Paciltexel Injection

Generic equivalent of the antineoplastic agent Taxol

Various cancers

The FDA approved the company's abbreviated NDA for the drug (11/15)

SuperGen Inc.
(SUPG) and
MGI Pharma
Inc. (MOGN)

Dacogen

Decitabine for injection; a hypomethylating agent

Myelodysplastic syndromes

The companies completed their NDA submission to the FDA; the first section was submitted in June (11/1)

Threshold
Pharmaceuticals
Inc.*

Glufosfamide

Small molecule targeting abnormal glucose metabolism

Metastatic pancreatic cancer

The FDA granted fast-track status to the Phase III drug for treating unresectable, advanced disease previously treated with gemcitabine (11/15)

YM BioSciences
Inc. (Canada;
TSE:YM)

TheraCIM hR3

Humanized anti-epidermal growth factor receptor monoclonal antibody

Brain cancer

The FDA granted orphan designation to the product in that indication (11/30)

CARDIOVASCULAR

Encysive
Pharmaceuticals
Inc. (ENCY)

Thelin (sitaxsentan)

Small molecule designed to block endothelin

Pulmonary arterial hypertension

The FDA granted orphan designation to the product, which is in Phase III trials (11/11)

NitroMed
Inc. (NTMD)

BiDil

Nitric oxide-enhancing oral agent

Heart failure

Submitted A-HeFT data set to the FDA, which the agency wanted before submission of an NDA amendment (11/1)

CENTRAL NERVOUS SYSTEM

Neurocrine
Biosciences
Inc. (NBIX)

Indiplon

Modified-release form of a non-benzodiazapine agent that targets the GABA-A receptor

Insomnia

Submitted an NDA with the FDA; an NDA for the immediate-release form of the drug was filed in October (11/22)

Phytopharm
plc (UK; LSE:PYM)

Myogane (PYM50018)

Product based on Asian plant extract believed to have neuroprotective effects

Amyotrophic lateral sclerosis

The FDA granted fast-track status to the product, which is in Phase I trials (11/9)

MISCELLANEOUS

BioMarin
Pharmaceutical
Inc. (BMRN)

Aryplase

Galsulfase; an enzyme- replacement therapy

Mucopoly- saccharidosis-VI

The company submitted a BLA with the FDA, seeking approval to market the drug for MPS-VI (11/29)

CuraGen
Corp. (CRGN)

CR002

Fully human monoclonal antibody that targets platelet-derived growth factor-D

IgA nephropathy

The FDA granted orphan designation for slowing the progression of IgA nephropathy and delaying kidney failure (11/11)

ESP Pharma
Inc.*

Terlipressin

Analogue of vasopressin delivered intravenously

Type I hepato-renal syndrome

The FDA granted orphan designation to the drug in that indication (11/10)

Nastech
Pharmaceutical
Co. Inc. (NSTK)

Nascobal

Nasally delivered formulation of cyanocobalamin

Vitamin B-12 deficiency

The FDA issued an approvable letter saying only inspection of a manufacturing facility and finalization of labeling are needed prior to approval (11/1)

Oragenics
Inc.*

--

Replacement therapy using a genetically modified strain of S. mutans

Dental caries

The FDA lifted a clinical hold it had placed on the product; a Phase I trial is planned for early 2005 (11/30)

Notes:

* Privately held.

BLA = Biologics license application; FDA = Food and Drug Administration; IND = Investigational new drug application; NDA = New drug application; SPA = Special protocol assessment.

Unless otherwise noted, the stock symbols listed for public companies are on the Nasdaq market.

LSE = London Stock Exchange; NYSE = New York Stock Exchange; TSE = Toronto Stock Exchange.