Pharmaceutical Processing World

  • Home
  • Regulatory
    • Recalls
  • Pharmaceutical Processing
  • Facility
  • Supply Chain
  • Equipment and Materials
  • Contract Manufacturing
  • Advertise
  • SUBSCRIBE

FDA Grants Orphan Drug Designation for 3,4-DAP for LEMS (Lambert EatonMyasthenic Syndrome) LEMS

By Pharmaceutical Processing | November 30, 2009

BioMarin PharmaceuticalInc. announced today that the FDA has granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP),amifampridine phosphate, for the rare autoimmune disease Lambert EatonMyasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drugdesignation in the E.U. Also, in October 2009, the Committee for MedicinalProducts for Human Use of the European Medicines Evaluations Agency adopted apositive opinion recommending approval of amifampridine phosphate for LEMS.If approved by the European Commission, amifampridine phosphate will be thefirst approved treatment for LEMS, thereby conferring orphan drug protectionand providing ten years of market exclusivity in Europe. “With its experience in the development and commercialization of orphandrugs, BioMarin is well-positioned to seek U.S. registration of amifampridinephosphate for LEMS, a serious and debilitating autoimmune disease oftenassociated with small cell lung cancer,” said Jean-Jacques Bienaime, ChiefExecutive Officer of BioMarin. “We look forward to meeting with the FDA inearly 2010 to determine the necessary regulatory path for amifampridinephosphate in the U.S. We are also preparing to launch the product in Europein the first quarter of 2010, and will also evaluate the best developmentstrategy for amifampridine phosphate in other indications in the U.S. andEurope.”

Related Articles Read More >

Doctor, woman patient and tablet for consulting with results, medical info and talk for healthcare with mockup space. Japanese medic, digital touchscreen or show video for surgery, wellness or advice.
Putting patients first in clinical trials
Confidently navigate the transition from bench to batch
Merck
FDA approves Merck’s Winrevair to treat pulmonary arterial hypertension
kobayashi pharmaceutical logo
Report: Japan health authorities investigate Kobayashi Pharmaceutical factory after five deaths
“ppw
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest news, technologies, and developments in Pharmaceutical Processing.

DeviceTalks Tuesdays

DeviceTalks Tuesdays

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.
Pharmaceutical Processing World
  • Subscribe to our E-Newsletter
  • Contact Us
  • About Us
  • R&D World
  • Drug Delivery Business News
  • Drug Discovery & Development
  • DeviceTalks
  • MassDevice
  • Medical Design & Outsourcing
  • MEDICAL TUBING + EXTRUSION
  • Medical Design Sourcing
  • Medtech100 Index
  • R&D 100 Awards

Copyright © 2025 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Pharmaceutical Processing World

  • Home
  • Regulatory
    • Recalls
  • Pharmaceutical Processing
  • Facility
  • Supply Chain
  • Equipment and Materials
  • Contract Manufacturing
  • Advertise
  • SUBSCRIBE