Company*
(Country;

Symbol)

Product

Description

Indication

Status (Date)


AUTOIMMUNE

Abbott
Laboratories

Humira (FDA-approved)

Adalimumab; fully human antibody designed to block TNF-alpha

Rheumatoid arthritis

FDA approved label expansion to include improvement in physical function for adults with moderately to severely active RA (8/10)

CANCER

Allos
Therapeutics

Inc. (ALLP)

Efaproxyn (efaproxiral; RSR13)

Small molecule designed to sensitize hypoxic areas of tumors prior to radiation

Brain metastases

The FDA granted orphan status to the drug for use as an adjunct to whole-brain radiation therapy for brain metastases in patients with breast cancer (8/3)

Aptamera Inc.*

AGRO100

Anti-nucleolin aptamer

Pancreatic cancer

The FDA granted orphan designation to the product in that indication, for which a Phase II study is being planned (8/27)

BioCryst
Pharmaceuticals
Inc.
(BCRX)

Forodesine hydrochloride (BCX-1777)

Purine nucleoside phosphorylase inhibitor; blocks T-cell synthesis

Chronic lymphocytic leukemia and acute lymphoblastic leukemia

The FDA granted orphan designation to the product in CLL and related leukemias, and in ALL; in February it got orphan status in T-cell non-Hodgkin's lymphoma (8/24)

Genmab A/S
(Denmark; CSE:GEN)

HuMax-CD4

Human antibody that targets the CD4 receptor on T lymphocytes

Mycosis fungoides

The FDA granted the drug orphan designation in that indication, which affects 75% of patients with cutaneous T-cell lymphoma (8/23)

ILEX Oncology Inc. (ILXO)

Clofarabine

Second-generation purine nucleoside analogue

Refractory/relapsed acute leukemia in children

Company provided FDA with new patient data to support the NDA, which was filed in March; the fil- ing moved the FDA action date back to Dec. 30 (8/24)

Medarex Inc.
(MEDX)

MDX-010
and MDX-1379

Fully human anti-CTLA-4 antibody; gp100 melanoma peptide vaccine

Metastatic melanoma

Reached agreement with FDA on a special protocol assessment for a pivotal Phase III trial of the drug combination in patients who have failed first-line therapy (8/23)

OSI
Pharmceutcials

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

Tarceva

Erlotinib HCl; small-molecule HER1/EGFR inhibitor

Non-small-cell lung cancer

The companies completed their NDA submission, seeking approval in advanced patients for whom chemotherapy has failed (8/2)

Seattle Genetics
Inc.
(SGEN)

SGN-40

Humanized monoclonal antibody that targets the CD40 antigen

Multiple myeloma

The FDA granted orphan status in that indication, for which the drug is in a Phase I trial (8/24)

CARDIOVASCULAR

Medicure Inc.
(Canada; TSE:MPH)

MC-4232

A combination MC-1 and and ACE inhibitor

Hypertension

Began Phase II trial in Canada to assess effects on 120 patients with co-existing diabetes and hypertension (8/11)

Myogen Inc.
(MYOG)

Ambrisentan

Type-A selective endothelin receptor antagonist

Pulmonary arterial hypertension

The FDA granted orphan designation to the drug in PAH, in which it is in two pivotal trials (8/3)

CENTRAL NERVOUS SYSTEM

Corcept
Therapeutics
Inc.
(CORT)

Corlux (mifepristone)

Oral GR-II antagonist with potential mitigating effects on cortisol

Psychotic major depression

Reached agreement with FDA on special protocol assessment for two pivotal trials; the first trial, Corcept 07, began immediately and will enroll up to 280 patients; the second trial, Corcept 06 to start in 4Q:04, will enroll about 440 patients (8/30)

New River
Pharmaceuticals

Inc.
(NRPH)

NRP104

Prodrug of amphetamine

Cocaine dependence

The preclinical product was granted fast-track status by the FDA (8/31)

Pharmos
Corp.
(PARS)

Dexanabinol

A tricyclic dextro-cannabinoid; synthetic analogue of marijuana

Severe traumatic brain injury

The FDA granted the drug orphan designation in that indication, for which Phase III results are expected in 4Q:04 (8/12)

INFECTION

Gilead Sciences Inc. (GILD)

Truvada

Fixed-dose combination of Gilead's approved HIV drugs Emtriva and Viread

HIV

The FDA granted accelerated approval of Truvada for use once daily in combination with other antiretroviral agents (8/2)

MISCELLANEOUS

Eyetech
Pharmaceuticals

Inc. (EYET) and Pfizer Inc.

Macugen

Pegaptanib sodium injection; pegylated anti-VEGF aptamer

Age-related macular degeneration

FDA accepted for review the NDA on the drug, the filing on which was completed in June (8/17); FDA panel discussed application but did not conduct a formal vote (8/27)

ISTA
Pharmaceuticals

Inc.
(ISTA)

Vitrase (FDA-approved)

Ovine hyaluronidase formulation

Use as a spreading agent

ISTA filed a supplemental NDA seeking approval for a smaller vial size (8/2)

Vital
Therapies Inc.*

--

Immortalized human liver cells

Acute liver failure

The FDA granted the product orphan designation in that indication (8/20)


Notes:

* Privately held

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

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

CSE = Copenhagen Stock Exchange; NYSE = New York Stock Exchange; TSE = Toronto Stock Exchange.

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