Acadia Pharmaceuticals has resubmitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of Nuplazid (pimavanserin) for hallucinations and delusions associated with psychosis in Alzheimer’s disease. The announcement from Acadia comes less than a year after the FDA rejected the company’s original application for Nuplazid to treat the broader category of dementia-related…
Conditions
Conditions
FDA Designates CT103A, CAR T-cell Therapy, an Orphan Drug
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to CT103A, an experimental CAR T-cell therapy that IASO Biotherapeutics and Innovent Biologics are developing to treat relapsed or refractory multiple myeloma. Orphan drug designation is given to therapies with a potential to substantially improve care for rare diseases, defined as conditions affecting…
More Studies Confirm Hefty Economic Burden of Rare Diseases
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion. Annie Kennedy is the chief of policy, advocacy, and patient engagement at the EveryLife Foundation for Rare Diseases. (Photo courtesy of the EveryLife Foundation) In…
Alzheimer’s Association Funds Research to Improve Lives of Caregivers
The Alzheimer’s Association is funding research that aims to improve the lives of caregivers as well as the dementia patients they serve. Amanda Leggett, PhD, a research assistant professor at the University of Michigan, has been awarded $150,000 by the U.S. nonprofit’s Dementia Care Provider Roundtable (AADCPR) to support an investigation into different caregiving styles.…
Anti-pTau Vaccine Induces Intended Immune Response in Ongoing Trial
ACI-35.030, an experimental anti-tau vaccine that AC Immune is developing for Alzheimer’s disease, was able to potently induce an immune response as intended and was generally well-tolerated, according to 10-week interim data from a Phase 1/2 clinical trial. “These latest interim results add to the robust clinical dataset supporting plans for continued late-stage development,” Andrea…
Q&A With RARE-X Disease Data Platform Founder, Nicole Boice
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of RARE-X, did with Global Genes. That…
Older Adults Aware of Alzheimer’s, But Know Little About Aduhelm
Although the vast majority of older Americans are concerned about Alzheimer’s disease, most are unaware of Aduhelm (aducanumab), the recently approved treatment for the disease, according to a survey study. “The contrast between older Americans who were very concerned about developing Alzheimer’s disease and those that actually knew anything about the drug was surprising,” Julie…
IMF Opening ‘Action’ Campaign to Mark Myeloma Awareness Month
The International Myeloma Foundation (IMF) is opening its annual Myeloma Action Month (MAM) campaign with a variety of events and undertakings to raise disease awareness across the community and the public-at-large. “Action” is theme of this year’s monthlong event, held each March. “The focus for this year’s Myeloma Action Month will be on actions that individuals…
Surge in Telehealth Brings Access, Convenience to Rare Disease Patients
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that burden, estimated at nearly $1 trillion,…
Fosgonimeton Shows Safety in Early Trial, Phase 3 Study Enrolling in US
Fosgonimeton, an experimental therapy Athira Pharma is developing to treat Alzheimer’s disease, was well-tolerated overall in a Phase 1 clinical trial that included Alzheimer’s patients as well as healthy volunteers. “These encouraging results showed a positive safety and tolerability profile of fosgonimeton across a wide dose range,” Hans Moebius, MD, PhD, Athira’s chief medical officer, said…
Cilta-cel – now Carvykti – Approved by FDA for Heavily-treated Myeloma
The U.S. Food and Drug Administration (FDA) has approved the CAR T-cell therapy Carvykti (ciltacabtagene autoleucel) — formerly known as cilta-cel — for certain adults with relapsed or refractory multiple myeloma. In particular, the approval makes Carvykti available for people who have received at least four prior lines of therapy, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD),…
Symposium Highlights Research, Care Disparities Among Latinos
Improved access to high-quality healthcare and research for Latinos living with Alzheimer’s disease and related dementia are needed, according to a summary report from the second Latinos & Alzheimer’s Symposium. Understanding underlying disease-related processes unique to Latino populations, and identifying social and environmental risk factors for this group also are required, the researchers noted. “By…