Reportedly, the US Congress had passed the Orphan Drug
Act in 1983 to provide incentives for industry investment in treatments for rare conditions.
Since 2007, the increase in orphan drug
spending has a direct link to the rising number of orphan drugs
that have since been approved by the Food and Drug Administration, according to Divino.
The Orphan Drug
Act (the Act) provides procedures to encourage and facilitate the development of drugs for rare diseases or conditions namely those that affect fewer than 200,000 persons in the U.
According to the company, the Orphan Drug
Designation is an important milestone for it and it recognises the promise of WTX101 as a potential new treatment for ALS.
The product has received orphan drug
designation for four indications: the treatment of invasive candidiasis, invasive aspergillosis, coccidioidomycosis, and rare mold infections caused by Scedosporium spp, Fusarium spp, and Mucorales fungi.
We wanted to determine whether the FDA requires that orphan drug
applications provide the conventional effectiveness data that are ordinarily expected for most drugs for more prevalent diseases or whether the agency has over the years exercised flexibility in approving drugs for patients with rare diseases.
designation in the European Union provides a 10-year period of post-approval marketing exclusivity and other financial incentives for new drugs that offer significant therapeutic advantage over other approved treatments of rare conditions (conditions affecting no more than 5 in 10,000 persons).
The orphan drug
and balloon technologies discussed here illustrate the significant impact that very expensive, very low volume technologies and high volume, "cost saving" technologies might have on an individual plan.
BioGaia (STO:BIOGB) announced on Monday that its subsidiary Infant Bacterial Therapeutics (IBT) has obtained the Orphan Drug
Designation for the prevention of necrotising enterocolitis (NEC) in Europe.
But companies that produce them under the orphan drug
designation are offered several advantages, including research grants, tax benefits, protocol assistance, marketing exclusivity (seven years in the United States) and regulatory challenges (such as a smaller number of clinical trials) that are less daunting than those faced by other new medicines.
Our MPS-VI program builds on BioMarin's expertise in the development of enzyme replacement therapies, and positions us now with two orphan drug
products in human clinical trials.