orphan drug status | GenomeWeb

orphan drug status

Non-small cell lung cancer patients with EGFR mutations now have three treatment choices, and AstraZeneca is working on newer personalized treatment options.

Pelago will license its cellular thermal shift assay technology to OmicScouts and the firms will collaborate to offer drug target identification and validation.

Entrectinib is Ignyta's lead product that the biotech is investigating in patients with tumors characterized by TrkA, TrkB, TrkC, ROS1, and ALK markers.

Alnylam Pharmaceuticals said this week that the US Food and Drug Administration has given orphan drug status to the company's investigational transthyretin-mediated amyloidosis ALN-TTR02 as a treatment for familial amyloidotic polyneuropathy, a common manifestation of the disease

Specifically, the designation is for the use of the drug against familial amyloidotic polyneuropathy, one of the predominant forms of the disease, Alnylam said.

FAP is an inherited, colorectal cancer syndrome characterized by the growth of colorectal polyps. Though the polyps are initially benign, they become malignant in nearly all cases in the absence of colectomy, according to Marina.

The provisions for the EU orphan designation provide incentives to companies developing orphan drugs, including the potential for up to 10 years of market exclusivity and various fee reductions for certain regulatory activities, Quark said.

The designation allows for possible research and development incentives, and qualifies the company for tax credits and a period of market exclusivity following market approval.

In Science this week: metagenomic-based technique for determining protein structure, and more.

An academic laments the rise of narcissism in the sciences, the Guardian reports.

Outgoing FDA commissioner Robert Califf writes in an editorial that the agency can help boost innovation.

The Trump transition team has asked NIH Director Francis Collins to remain at his post, though it's unclear for how long that will be.