Cysteamine--Brands, Cost, Availability


Cystagon
http://medlibrary.org/lib/rx/meds/cystagon/page/4/

 Approximate cost: $8,000 per year

 CVS CAREMARK Pharmacy, in partnership with Mylan Pharmaceuticals, is currently the exclusive pharmacy distributor for Cystagon capsules. Cystagon has been approved by the U.S. FDA for the management of nephrotic cystinosis in children and adults.

 Cystagon is available in 
50 mg oral capsules
150 mg oral capsules 

 To find out more about CVS Caremark call 1-800-238-7828.
https://www.caremark.com/wps/portal 



Cystagon® is a registered trademark of Mylan Pharmaceuticals Inc.

Procysbi
http://www.procysbi.com/

Approximate cost: $250,000 per year

PROCYSBI is a delayed-release form of cysteamine bitartrate that works by continuously reducing the toxic levels of cystine in the cells. This may help prevent irreversible cellular damage. PROCYSBI can provide continuous control of cystine when taken every 12 hours. 

 Prescribing information: http://www.procysbi.com/docs/Procysbi-Full-Prescribing-Information.pdf 
Lynovex

  • An oral (gel capsule) form of cysteamine (not yet available)

  • In addition to the oral formulation to treat acute exacerbations in cystic fibrosis, NovaBiotics has formulated dry powder inhalable forms as a maintenance therapy for chronic use. This form of targeted delivery is being developed in order to deliver maximum efficiency locally within the lung.


NM001(Lynovex®) is a first-in-class orphan drug candidate for cystic fibrosis, an inherited life-limiting disease which affects the lungs, digestive tract and other organ systems.

NM001 is unique, providing the first of its kind treatment for cystic fibrosis (and also COPD and other chronic respiratory conditions), as a single treatment with multi-functionality it is able to; breakdown the excessive mucus produced by the lining of the airways in patients, kill the bacteria responsible for the recurrent respiratory infections and disrupt the biofilms in which they colonise. NM001 therefore has the potential to improve lung function and reduce or even prevent degenerative, long-term respiratory tract damage in patients.

Clinical and Commercial Development
An oral (gel capsule) form of NM001 is poised to undergo Phase I/IIa clinical trials in Q2/2014 to assess its potential as a treatment for acute exacerbation events in cystic fibrosis patients. This work is supported by the Cystic Fibrosis Trust and Health Sciences Scotland.
NovaBiotics is working in collaboration with NHS Grampian and the University of Aberdeen’s Cystic Fibrosis research team to undertake initial clinical evaluation and trials.
Although NM001 is in its early stages of development for cystic fibrosis, it is an orphan drug candidate and its API is already approved for use in an unrelated orphan metabolic disease (and has been shown to be safe and well tolerated), therefore NM001 (Lynovex®) has the potential for fast-track clinical development.
Molecule Class and Formulation
The API of NM001 (Lynovex®) is cysteamine.
In addition to developing NM001 as an oral formulation to treat acute exacerbations in cystic fibrosis, NovaBiotics has formulated dry powder inhalable forms as a maintenance therapy for chronic use. This form of targeted delivery is being developed in order to deliver maximum efficiency locally within the lung.
Mode of Action
NM001 (Lynovex®) has a unique, dual antibacterial-mucoactive mode of action which aggressively tackles both of the major clinical features responsible for progressive lung deterioration in cystic fibrosis.
NM001 not only kills Gram-negative and Gram-positive respiratory bacteria (including Pseudomonas aeruginosa and Burkholderia cepacia) but prevents and disrupts formation of the slimy biofilms which these pathogens create in order to establish long-term, antibiotic insensitive infections/colonies in the cystic fibrosis lung environment. Experiments with sputum from cystic fibrosis patients have demonstrated NM001 is active against the panel of multi-drug resistant cystic fibrosis pathogens within the complex and challenging matrix of cystic fibrosis sputum and that NM001 reverses resistance/insensitivity to antibiotics in these microbes.
NM001 (Lynovex®) is also mucolytic; acting to disrupt the mucus in cystic fibrosis patients’ airways. In vitro and ex vivo tests have shown NM001 to be more effective than currently available mucoactive agents.

Lynovex Presentation with charts and graphs

No comments:

Post a Comment

Subscribe to: Posts (Atom)