This week's Lab Notes has items on a Philadelphia biotech company's approval for a combination therapy targeting a rare disease, a key designation for another firm's experimental therapy for the same ailment, a new CEO appointment and more.
Here's the roundup:
Amicus Therapeutics (NASDAQ: FOLD)
The Philadelphia biotech company received Food and Drug Administration approval for its two-component Pompe disease therapy.
The treatment that combines Pombiliti and Opfolda is indicated for adults living with late-onset Pompe disease who are not improving on their current enzyme replacement therapy.
Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase. Reduced levels of the enzyme lead to the accumulation of the substrate glycogen in the lysosomes of muscle cells, and glycogen buildup causes muscle damage.
In the United States, an estimated one person in 40,000 is affected by Pompe disease. The global Pompe disease market size in 2021 was estimated at $1.4 billion by Emergent Research.
John F. Crowley, executive chairman of Amicus (NASDAQ: FOLD), called the FDA's approval of Pombiliti and Opfolda "a testament to the power of science, medicine, and our passionate determination to improve the lives of people living with Pompe disease."
Amicus said it will launch the therapy in the U.S. immediately.
Aro Biotherapeutics
The Philadelphia biotechnology company specializing in tissue-targeted genetic medicines was granted rare pediatric drug designation from the FDA for ABX1100, its experimental Pompe disease treatment.
“Pompe Disease affects patients of all ages but can be particularly devastating in children,” said Dr. Mittie Doyle, Aro's chief medical officer.
ABX1100 is designed to treat Pompe disease by reducing glycogen buildup in the muscles.
The FDA awards rare pediatric disease designation to drugs treating rare serious or life-threatening diseases that affect patients from birth to 18 years of age. Companies that receive the designation are entitled to receive a Pediatric Review Voucher, upon approval in a pediatric disease indication, giving the holder access to a priority review, reduced review time and potential expedited product approval. The vouchers can, and have, been sold to other drug developers.
ChromaTan
The privately held bioprocessing and bio-tools company based at the Spring House Innovation Park in Lower Gwynedd named Dr. Rajiv Datar as its president and CEO.
Datar has more than 25 years of management experience in the life sciences industry having most recently served as co-founder and CEO of Propelle Therapeutics.
ChromaTan's previous CEO, company founder Oleg Shinkazh, is staying on as chief technology officer and will focus on advancing the field of integrated continuous bioprocessing.
Founded in 2007, the 20-person company has created Kascadee, a novel purification system based on continuous countercurrent tangential chromatography, which has demonstrated the ability to cut in half the cost of biologics purification while offering significantly higher yields for gene therapies.
ChromaTan’s customer list includes pharmaceutical giants Merck, Roche, and Takeda along with several startups.
Strados Labs
The Philadelphia medical device company has formed a partnership with Ann & Robert H. Lurie Children’s Hospital of Chicago for a clinical trial to evaluate the effectiveness of Strados' RESP Biosensor in children with wheezing symptoms.
The RESP Biosensor, approved for use in adults, is wearable device used to remotely monitor lung sounds for asthma symptoms such as coughing and wheezing.
Pediatric asthma affects more than 5 million children each year with one in six children visiting the emergency room annually because of complications, according to the Centers for Disease Control and Prevention.
Quick Hits
Camden-based Cooper University Health Care’s Center for Innovation has received a $100,000 grant from the Foundation for Health Advancement to support the development of a medical device invented by two Cooper Health doctors that is designed to aid physicians performing cardiac catheterizations and related procedures by helping them overcome challenges accessing the femoral artery. The Antegrade Femoral Artery Entry Device and Sheath was invented and developed by vascular and endovascular surgeons Dr. Jeffrey P. Carpenter, chairman and chief of the department of surgery at Cooper, and Dr. Katherine K. McMackin, director of vascular surgical research at Cooper. The grant funding will be used for the development and testing of several prototype devices.… Tom Malecki has joined The National Disease Research Interchange of Philadelphia as its new CFO and vice president of administration. Malecki was previously the COO and CFO at the Utility Emergency Services Fund, a Philadelphia nonprofit dedicated to assisting families facing difficulties finding stable housing.
