Rare disease drug development companies battle with economic challenges and small patient populations, but new technology alongside a human connection are helping researchers and marketers identify patients, educate physicians and build networks. At Pharma USA, a Reuters Events meeting in Philadelphia March 16-17, executives from Astrazeneca plc and UCB SA discussed their successes with rare disease launches, presenting their playbooks for building trust and driving adoption among patients.
The regulatory clouds that have been darkening the U.S. FDA landscape of late for Uniqure NV’s gene therapy AMT-130 in Huntington’s disease may be parting a bit with the announced departure of Vinay Prasad as director of the agency’s CBER at the end of April.
At the current pace of innovation in the U.S. rare disease space, developing and approving therapies for just half of the 10,000-plus known rare diseases would take more than 160 years, Bradley Campbell, president and CEO of Amicus Therapeutics Inc., recently told the Senate Committee on Aging.
A lot of distance lies between talking regulatory flexibility and actually being flexible. That message was driven home again after Uniqure NV disclosed in its latest earnings report March 2 that the U.S. FDA wants a sham-controlled study before it will consider approval of the company’s gene therapy AMT-130 in Huntington’s, a rare disease currently affecting about 41,000 people in the U.S.
Another phase III failure of Theravance Biopharma Inc.’s norepinephrine reuptake inhibitor ampreloxetine to treat symptomatic neurogenic orthostatic hypotension, this time in patients with the rare disease multiple system atrophy, means the end of the company’s R&D efforts. Cutting its workforce in half and terminating all development of ampreloxetine, its only pipeline product, the Dublin-based company’s shares (NASDAQ:TBPH) sank by 26%, or $4.99, to close March 3 at $13.96.
While the annual State of the Union address has morphed over the years from a summation of the state of the U.S. government and the president’s legislative agenda into political theater on both sides of the aisle, President Donald Trump did include some recommendations to Congress in his Feb. 24 speech. Among those recommendations was a request for Congress to codify his most-favored-nation pricing policy for prescription drugs.
Following a clinical hold last October of Intellia Therapeutics Inc.’s Magnitude and Magnitude-2 phase III trials of CRISPR/Cas9 gene editing therapy nexiguran ziclumeran (nex-z) to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively, the U.S. FDA lifted the hold on Magnitude-2, pushing the company’s shares up by 22% in early trading Jan. 27.
After selling off amyotrophic lateral sclerosis therapeutics last month, Tanabe Pharma America Inc. emerged a forerunner in another rare disease space with positive top-line phase III data of dersimelagon (MT-7117) for erythropoietic protoporphyria/X-linked protoporphyria.
After selling off amyotrophic lateral sclerosis therapeutics last month, Tanabe Pharma America Inc. emerged a forerunner in another rare disease space with positive top-line phase III data of dersimelagon (MT-7117) for erythropoietic protoporphyria/X-linked protoporphyria.
The U.S. FDA has approved Zycubo (copper histidinate) as the first treatment for Menkes disease, a rare, genetic disease affecting children who cannot absorb copper through their intestines, leading to seizures, weak muscles, a failure to thrive and, ultimately, if left untreated, an early death by age 3.
