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ORPHAN DISEASE SERIES - Glaucoma: Can Orphan-Drug Status Offer an Entry into a Mature Market?

Authors
Matthew Drapeau
Jennifer Moniz Carpenter, M.B.A.
Yimin Lou, M.Sc., M.A.
Francis Sullivan
Spectrum -- June 2008

  Introduction:

The glaucoma market has not seen any new mechanisms for treatment since the launch of prostaglandin analogues in the mid 1990s. Despite the efficacy of existing agents, glaucoma treatment still exhibits a significant level of unmet need, especially to address unreliable patient compliance and unacceptable side effects. Additionally, several orphan conditions exist that include glaucoma as a relevant symptom. Focusing on any of these areas could provide an entry for companies looking to emerge in this market.

  Get the Answers You Need to Shape Your Strategy:

Various forms of glaucoma affect more than 50 million people in the United States; the majority are treated with agents to lower intraocular pressure. What unmet needs exist that could provide entry for novel agents? What agents are currently in development? What do thought leaders say they would like to see in an emerging treatment?

Ophthalmologists interviewed say that compliance is one of the biggest issues in the successful treatment of glaucoma. What do these thought leaders say about their prescribing preferences related to dosing regimens? What novel agents are in development to test new delivery methods and new formulations?

Several companies are developing agents to improve existing therapy options. What early clinical data have been released regarding these studies? How do thought leaders view the potential of these emerging drugs?

A recent search of the National Institute of Health’s Office of Rare Diseases revealed more than 20 orphan conditions that include glaucoma as a relevant symptom. What indications would benefit from orphan-drug discovery? Which agents have already received orphan-drug designation for glaucoma treatment?

  Scope:

Current treatments for glaucoma: beta blockers; prostaglandin F2 analogues; carbonic anhydrase inhibitors; alpha 2-adrenoreceptor agonists; combination agents.

Epidemiology: prevalent cases of primary open-angle and congenital glaucoma (CG); expected growth through 2017.

Overview of glaucoma: anatomy of the eye; types of glaucoma; risk factors for glaucoma; diagnosis, disease progression, prognosis.

Emerging treatments for glaucoma: Alcon’s anecortave acetate; QLT’s latanoprost punctal-plug delivery system; Santen/UBE Industries’ DE-104.

Glaucoma as an orphan indication: neovascular glaucoma; juvenile primary open-angle glaucoma.

Glaucoma market outlook: decline in market due to patent expiry and lack of new branded agents; emerging agents with improved side-effect profiles and delivery mechanisms more likely to see significant uptake.

Orphan Disease Series: As competition increases in larger disease states, companies are turning to orphan diseases that offer substantial pricing advantages and lower regulatory hurdles because of high unmet need. The Orphan Disease Series provides critical market information, including disease prevalence, profiles of marketed and emerging agents, and insight into the prescribing choices of disease specialists for diseases with smaller patient populations. These reports will help you to maximize the potential of your agents in these challenging markets, including the regulatory benefits available and the potential for partnership with charitable organizations.

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Companies:

Aerie Pharmaceuticals

Alcon

Allergan

Barr Pharmaceuticals

Genentech

GeneTherapy

Inspire

Merck

Mitsubishi Tanabe

Mobius Therapeutics

Novartis

Pfizer

QLT

Sanofi-Aventis

Santen

Senyu

UBE Industries

Wyeth




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