This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here
TheStreet Open House

Alexion’s Soliris® (eculizumab) Receives Orphan Drug Designation For The Treatment Of Neuromyelitis Optica (NMO)

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that Soliris ® (eculizumab), the company’s first-in-class terminal complement inhibitor, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of neuromyelitis optica (NMO), a life-threatening, ultra-rare neurological disorder. In a Phase 2 study presented at the 2012 annual meeting of the American Neurological Association (ANA), Soliris treatment was associated with a significant reduction in the frequency of relapses (recurring attacks) in patients with severe, relapsing NMO. 1 Soliris is not approved for the treatment of patients with NMO.

The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.

“There are no approved therapies for patients with NMO, an extremely rare and debilitating disease that can lead to paralysis, blindness and death,” said Martin Mackay, Ph.D., executive vice president and global head of R&D at Alexion. “This orphan drug designation is a positive step toward understanding and meeting the needs of this underserved patient population. In clinical trials to date, terminal complement inhibition with Soliris appeared to significantly reduce the attack rate in patients with severe, refractory relapsing NMO.”

About NMO

In patients with NMO, uncontrolled complement activation causes destruction of myelin-producing cells, leading to severe damage to the central nervous system (CNS), including the spinal cord and optic nerve. 2-4 The disease leads to severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death. 5-7 Patients with NMO have a life-long exposure to the uncontrolled complement activation due to chronic autoimmune attack, and most patients experience an unpredictable, relapsing course of disease with cumulative disability, as each attack adds to the neurologic disability. 6,8,9 Fifty percent of relapsing NMO patients have been reported to sustain permanent severe disability, including paralysis and blindness, within five years of disease onset. 10 Most NMO-related deaths result from respiratory complications from NMO attacks. 10,11 The disease primarily affects women, with a female to male ratio as high as a 9:1. 12

Phase 2 Data in Patients with NMO

The Phase 2 study reported at the ANA 2012 annual meeting and subsequently published in The Lancet Neurology 13 was a single-arm, open-label, investigator-initiated trial in 14 women with severe, relapsing NMO who were treated for one year. The study met its primary efficacy endpoint, reduction in annualized relapse rate, with high degrees of clinical and statistical significance: a decline in the median annualized attack rate from 3.0 attacks per year pre-Soliris treatment to 0 attacks per year during 12 months of chronic Soliris treatment (p<0.0001). After 12 months of treatment, 86% (12 of 14) of these severely affected patients were completely attack-free. 1

Additionally, Soliris was associated with significant improvements in key secondary endpoints. The median expanded disability status scale (EDSS) score improved from 4.3 pre-treatment to 3.5 after 12 months of treatment with Soliris (p<0.01). Importantly, all patients experienced either improvement or stability in all key outcome measures, including EDSS, ambulatory function as measured by the Hauser Ambulation Index, and visual function as measured by visual acuity. Soliris was generally well-tolerated, with the three most common adverse events being headache, nausea, and dizziness. One case of meningococcal sepsis occurred. The patient made an uneventful recovery and restarted treatment with eculizumab to complete the study. 1

About Soliris

Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the U.S., E.U., Japan and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder characterized by complement-mediated hemolysis (destruction of red blood cells). Soliris is also approved in the U.S., E.U., and other countries as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS), a genetic, life-threatening, ultra-rare disease characterized by complement-mediated thrombotic microangiopathy (TMA, the formation of blood clots in small vessels).

1 of 4

Select the service that is right for you!

COMPARE ALL SERVICES
Action Alerts PLUS
Try it NOW

Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
  • Weekly roundups
TheStreet Quant Ratings
Try it NOW
Only $49.95/yr

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
  • Upgrade/downgrade alerts
Stocks Under $10
Try it NOW

David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
  • Weekly roundups
Dividend Stock Advisor
Try it NOW

Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Alerts when market news affect the portfolio
  • Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
Real Money Pro
Try it NOW

All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.

Product Features:
  • Real Money + Doug Kass Plus 15 more Wall Street Pros
  • Intraday commentary & news
  • Ultra-actionable trading ideas
Options Profits
Try it NOW

Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • 100+ monthly options trading ideas
  • Actionable options commentary & news
  • Real-time trading community
  • Options TV
To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.
Submit an article to us!
DOW 17,293.58 +27.59 0.16%
S&P 500 2,010.34 -1.02 -0.05%
NASDAQ 4,575.5810 -17.8440 -0.39%

Brokerage Partners

Rates from Bankrate.com

  • Mortgage
  • Credit Cards
  • Auto

Free Newsletters from TheStreet

My Subscriptions:

After the Bell

Before the Bell

Booyah! Newsletter

Midday Bell

TheStreet Top 10 Stories

Winners & Losers

Register for Newsletters
Top Rated Stocks Top Rated Funds Top Rated ETFs