This Day On The Street
Continue to site
ADVERTISEMENT
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

BioMarin Announces Decision To Start Phase 3 Program For PEG-PAL In 2Q 2013

"This Phase 2 study has shown that PEG-PAL appears to control Phe levels independent of the Phe-restricted diet and can produce a sustained response," stated Dr. Nicola Longo, Professor of Pediatrics and Adjunct Professor of Pathology at the University of Utah and Clinical Investigator in the PEG-PAL Phase 2 trial. "Most adult PKU patients are unable to adhere to a Phe-restricted diet and as a result, they can suffer from decreased mental, social and behavioral functioning. PEG-PAL may offer these patients the possibility of freedom from the Phe-restricted diet."

During the Phase 2 program, PEG-PAL was generally well-tolerated. Only two patients discontinued PEG-PAL due to adverse reactions, neither of which was reported to be severe. Twelve other patients discontinued participation in the program primarily due to logistical reasons. Forty-two patients remain on PEG-PAL in the open-label extension study. Of these, 25 patients have been treated longer than one year, including 15 patients who have been treated longer than two years. In total, there have been more than 800 patient months of treatment with PEG-PAL. The principal adverse reaction believed to be related to PEG-PAL occurs during initial dosing of the drug and appears to be hypersensitivity-type reactions consisting of injection-site reaction, disseminated skin reaction or joint pain. While these reactions were observed in nearly all PEG-PAL patients during their initial introduction to the drug, the reactions are generally mild to moderate and self-limited. Importantly, patients who have these types of reactions have all been successfully re-treated with PEG-PAL. During long-term exposure to PEG-PAL, injection-site and hypersensitivity reaction rates decreased dramatically and there was no laboratory evidence of liver or kidney injury.

Most recently, in the Phase 2 Part D study, BioMarin has investigated an accelerated personalized dosing regimen for introduction of PEG-PAL to get patients to active doses as quickly as possible while minimizing hypersensitivity-type reactions. Patients started with a fixed induction dose of 2.5 mg for four weeks. The dose escalated to 75 mg/week based on individual patient tolerability, at which point patients switched to a daily maintenance dose. In this part of the Phase 2 program, the earliest a patient was able to reach the maintenance dose was nine weeks, and half were able to achieve efficacy at or prior to reaching maintenance dosing  in less than 13 weeks. 

2 of 4

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

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
Stocks Under $10

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

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Only $9.95
14-Days Free
Submit an article to us!
SYM TRADE IT LAST %CHG

Markets

DOW 18,043.84 -26.56 -0.15%
S&P 500 2,105.46 -9.03 -0.43%
NASDAQ 4,971.9010 -45.0280 -0.90%

Partners Compare Online Brokers

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs