In ovarian cancer as well as melanoma, FANG has been given orphan drug status by the FDA. (See BioWorld Today, March 12, 2012.) "The reason we're being a little bit more aggressive in moving forward with submitting a 'breakthrough' application is that our Phase I data were also very encouraging," and could become important in the bid to get the vaccine approved as quickly as possible, Nemunaitis said

It has orphan drug designation in the U.S

It has orphan drug designation in the U.S

orphan drug designation 4/24/13 DIABETES pSivida Corp. (Watertown, Mass.) and Alimera Sciences Inc. (Atlanta) Iluvien Fluocinolone acetonide intravitreal implant Chronic diabetic macular edema Submitted its response to the FDA's second complete response letter 4/2/13 GASTROINTESTINAL Sucampo Pharmaceuticals Inc. (Bethesda, Md.) Amitiza Lubiprostone Opioid-induced constipation FDA approved it in patients with chronic, noncancer pain 4/24/13 INFECTION BioAlliance Pharma SA (Paris) Sitavig

said the European Commission granted orphan drug designation to multityrosine kinase inhibitor lenvatinib for the treatment of follicular and papillary thyroid cancer

Jennerex Biotherapeutics Inc., of San Francisco, said the FDA granted orphan drug status for Pexa-Vec (JX-594, pexastimogene devacirepvec) for the treatment of hepatocellular carcinoma (HCC

Eisai Inc., of Woodcliff Lake, N.J., said the European Commission granted orphan drug designation to multityrosine kinase inhibitor lenvatinib for the treatment of follicular and papillary thyroid cancer

Hyperion Therapeutics Inc., of South San Francisco, said the FDA notified the firm that Ravicti (glycerol phenylbutyrate) qualified for orphan drug exclusivity, giving the product exclusivity on the market for seven years from the date of approval on Feb. 1

Diabetes is another area of interest for Capital, Düster said, as well as the smaller or mid-cap companies developing orphan drugs

The FDA approved Cystaran in October 2012 and granted the product orphan drug status...On Tuesday, the FDA also approved Raptor Pharmaceutical Corp.'s orphan drug Procysbi (RP103, cysteamine bitartrate delayed-release) to treat the larger indication of nephropathic cystinosis. (See BioWorld Today, May 1, 2013

The FDA put some lift under the wings of Raptor Pharmaceutical Corp., approving the orphan drug Procysbi (RP103, cysteamine bitartrate delayed-release) on its PDUFA date in the initial indication of nephropathic cystinosis (NC...and $100,000 per patient per year ex-U.S., based on Raptor's comments suggesting a pricing premium to Cystagon and the inverse relationship between orphan drug pricing and patient prevalence

TikoMed AB, of Viken, Sweden, initiated a Phase I/II study of orphan drug candidate TM-400, designed to improve the outcome of hematopoietic stem cell transplantation in hematological cancers such as multiple myeloma and non-Hodgkins lymphoma

Last year alone orphan drug sales increased 7.1 percent to $83 billion from the previous year...Of the orphan drugs in the pipeline, the report indicates that elotuzumab (AbbVie/Bristol-Myers Squibb Co.) indicated in multiple myeloma is the most valuable.

As one session attendee was overheard explaining to another as they left the orphan drug session, Mendlein is a rare combination of PhD and JD

On the subject of orphan drugs, Weihong said, the CFDA is "still doing research about policy-making

Designed to alter the splicing of a closely related gene to increase production of fully functional SMN protein, ISIS-SMNRx was granted orphan drug status and fast-track designation in the U.S. Under the terms of a 2012 agreement, Isis is responsible for global development and Biogen has the option to license the compound until completion of the first successful Phase II/III study. (See BioWorld Today, Jan. 5, 2012

The main options are to take Cx611 forward into a Phase IIb trial in refractory rheumatoid arthritis, do a Phase IIb in a less-difficult-to-treat rheumatoid arthritis population, or to segue into a study in lupus or another autoimmune disease where it would be possible to get orphan drug status

The main options are to take Cx611 forward into a Phase IIb trial in refractory rheumatoid arthritis, do a Phase IIb in a less-difficult-to-treat rheumatoid arthritis population, or to segue into a study in lupus or another autoimmune disease where it would be possible to get orphan drug status

This surge in R&D on orphan drugs will fuel a rapid growth in the worldwide orphan drug market, which is predicted to hit $127 billion by 2018, accounting for nearly 16 percent of total prescription drug sales...Among the orphan drugs in the pipeline, the report contended that elotuzumab (AbbVie, Bristol-Myers Squibb Co.) indicated in multiple myeloma is the most valuable.