The Meveol designated orphan drug in Europe and the United States.

The Designation orphan drug is granted to products that are designed to treat orphan diseases (less than 5 per 10 000 people in the European Union). After counter-analysis of a folder by experts from the EMEA (Europe), FDA (USA) the product can be designated an orphan drug if it can demonstrate a potential therapeutic efficacy superior existing drugs.

The potential drug can be recognized if the orphan drug clinical trials with patients confirmed these results.

The OSCN / Lactoferrin, developed under the name of Meveol is appointed Mr.orphan medicinal product (potential) By the EMEA and FDA. Efficacy results have convinced experts of its major therapeutic potential in treating respiratory infections resistant to most cystic fibrosis.

Conclusion - EMEA designation

More:

What is an orphan?

Who are the independent experts who give the orphan drug designation?

List of designated orphan drug for cystic fibrosis in Europe since the introduction of the device in 2000.

What is an orphan?

In Europe, between 25 and 30 million people suffering from rare diseases have no treatment. Despite obvious needs, the development of drugs is not easy to promote ...

There are so rare disease that often it is not profitable for industry to develop treatments. Sales of a drug for a rare disease would never be sufficient to offset the cost of development and marketing.

What is an orphan?

As defined by the European Agency for the Evaluation of Medicinal Products, orphan drugs are intended for diagnosis, prevention or treatment of rare diseases, causing very serious or life threatening. A disease is considered rare when it does no more than five cases per ten thousand persons in the European Union. This scarcity is such that pharmaceutical companies are reluctant to develop drugs whose cost of placing on the market can be offset by the expected sales in the absence of incentives. Indeed, the process of developing a new molecule discovery to commercialization is long, extremely expensive and uncertain.

According Orphanet, an information server on rare diseases, it is certainly not profitable for industry to develop these orphan drugs but it is a genuine public health need. Our society can not do without the solidarity towards all patients, their disease is rare or not! This association also distinguished the case of orphan indications where a substance can be used to treat a common disease but not developed in another indication rarer.

How to reconcile public health and market forces?

Orphanet distinguishes three cases:

Products intended to treat rare diseases affecting less than one in 2000 in Europe: These treatments are designed to treat patients with serious illnesses for which there is no treatment option;

The recalled products for economic reasons or therapeutic : One example is thalidomide used as a hypnotic before being withdrawn from the market because of the teratogenic (causing birth defects). However, its anti-inflammatory and immunomodulatory were interesting in diseases such as leprosy or lupus erythematosus;

Products not developed : Because they are from a non-patentable, and those concerning contracts not be met, certain avenues of research are not exploited.

Not to let on the road thousands of patients, the government has introduced incentives for industrial health and biotechnology.

How to promote development of orphan drugs?

Governments and associations have underlined the need to develop economic incentives to encourage pharmaceutical companies to develop and market orphan drugs. The first country to have implemented these measures is the United States in 1983 with the Orphan Drug Act. In the 90s, Japan and Australia have enacted legislation on orphan drugs. It was only in 1999 that Europe followed suit.

The European Parliament and the Council adopted a regulation that encourages the pharmaceutical industry to develop and market orphan drugs. Tax incentives, protocol assistance, exclusive of 10 years, etc.. These measures are part of "accompanying". While little visible progress, but fundamental to the sick.

Luke Blanchot

Sources: Orphanet Eurorsis Alliance Rare Diseases

Definition from the website doctissimo.fr with the permission of the webmaster.

Who are the independent experts to give the orphan drug designation?

In Europe, the EMEA, European Medicine Agency, a committee is specifically responsible for assessing applications for Orphan Drug designation. This is the COMP is consititué of 39 independent experts, including one representative from each European Union country.

COMP role:http://www.ema.europa.eu/htms/general/contacts/COMP/COMP.html

The applicant submits a case in which he must provide evidence of efficacy superior to existing drugs.

The record is vetted by experts, which all the additional explanations necessary before submitting the case to the COMP to the plenary session, which may require an examination in the presence of the applicant.

List of members of the Committee of the EMEA Orphan Drug: http://www.ema.europa.eu/htms/general/contacts/COMP/COMP_members.html

List of Drug Substances (potential) orphan for cystic fibrosis in Europe since the establishment of dispositifi in 2000.

Substances

• (3 - [5 - (2-fluoro-phenyl) - [1,2,4] oxadiazole-3-yl]-benzoic acid
• Alginate oligosaccharide (G-block) fragment
• Alpha-1 antitrypsin (inhalation use)
• Alpha-1 proteinase inhibitor (inhalation use)
• Amikacin sulfate (liposomal)
• Avian IgY polyclonal antibody Against Pseudomonas aeruginosa
• Aztreonam lysinate (inhalation use)
• Bacterial lipase
• Ciprofloxacin (liposomal)
• Ciprofloxacin (inhalation)
• Colistimethate sodium
• Denufosol tetrasodium
• Dexamethasone sodium phosphate encapsulated in human erythrocytes
• Dornase alfa
• Duramycin
• Glutathione
• Heparin sodium
• Hypothiocyanite / lactoferrin
• Recombinant protein inhibitor of human leukocyte elastase
• L-Lysine-N-acetyl-L-cysteinate
• Levofloxacin hemihydrate
• Mannitolum
• N-(2,4-Di-tert-butyl-5-hydroxyphenyl) -1,4-dihydro-4-carboxamide-3-OXOQUINOLINE
• Pancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase)
• Recombinant dog gastric lipase
• Recombinant human bile salt-stimulated lipase
• Recombinant microbial lipase
• Temocillin sodium
• Tobramycin (inhalation powder)
• Tobramycin (inhalation solution)
• Tobramycin (liposomal)
• Tobramycin (inhalation)
• Source: orphanet.

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