The FDA’s approval was based on clinical data from the global Phase II PaTH Forward and Phase III PaTHway trials. Dr. Lynn Kohlmeier, an investigator in the PaTHway Trial, emphasized the importance of this approval, stating, “The ability to address the underlying cause of this disease is crucial and will be an important advancement for our patients with hypoparathyroidism.”
Ascendis plans to launch YORVIPATH with a suite of patient services through its U.S. Ascendis Signature Access Program (A.S.A.P.), offering support and financial assistance. The company anticipates initial supply availability by the first quarter of 2025 and aims to commercialize existing manufactured product by the fourth quarter of 2024, pending FDA approval.
ARS Pharma Gets FDA Green Light for First Nasal Spray for Severe Allergic Reactions
ARS Pharmaceuticals, Inc. made an announcement that the FDA has approved neffy (epinephrine nasal spray) 2 mg for treating Type I Allergic Reactions, including anaphylaxis, in adults and children weighing ≥30 kg (66 lbs). This approval marks the first significant innovation in epinephrine delivery in over 35 years and introduces the first needle-free option for managing severe allergic reactions.
Dr. Thomas B. Casale, from the University of South Florida, highlighted the importance of this development: “Until today, patients with severe allergic reactions only had one treatment option – an often painful and anxiety-inducing needle injection of epinephrine. FDA approval of neffy means that patients finally gain a long-awaited, needle-free, easy-to-carry epinephrine delivery method.”
Type I allergic reactions can lead to life-threatening anaphylaxis, resulting in approximately 500,000 emergency room visits annually, with nearly 60% of patients not receiving epinephrine before arriving at the ER. Richard Lowenthal, CEO of ARS Pharmaceuticals, emphasized the impact of this new treatment: “This approval marks a watershed moment in addressing an unmet medical need for people with Type I allergies – a treatment alternative that avoids the need to inject epinephrine with a needle.”
The FDA’s approval of neffy is based on data from five primary registration studies and several supportive studies. Dr. Jonathan Spergel from Children’s Hospital of Philadelphia noted, “We know that earlier administration is better, and for many, the needle is a barrier that causes dangerous hesitation. That is why the field has long pursued an effective treatment approach that does not require an injection.”
FDA Rejects Lykos’ Mdma-Assisted PTSD Therapy After Negative Adcomm
The FDA has declined to approve MDMA, commonly known as ecstasy, as a treatment for post-traumatic stress disorder (PTSD), Lykos Therapeutics announced on 9th Aug 2024. This decision is seen as a minor setback for the movement to use psychedelics in mental health treatments. The FDA’s decision followed an independent advisory committee’s recommendation against approval, citing insufficient evidence of the drug’s safety and effectiveness.
The advisory committee raised concerns about poorly designed studies, allegations of sexual misconduct during clinical trials, and potential health risks, including heart problems and abuse. A review by FDA scientists also noted issues with trial execution, such as the likelihood that patients and therapists could guess who received the medication versus the placebo.
Despite this rejection, experts remain optimistic about the future of psychedelic therapies. Holly Fernandez Lynch, an associate professor of medical ethics at the University of Pennsylvania, commented, “I think it will be a temporary setback. The advisory committee and FDA gave very clear indications of what they’re looking for in terms of study design and adverse event reporting.” She believes that Lykos and other companies now have clearer guidelines to achieve future approvals.
There are approximately four dozen MDMA trials in various stages of clinical development, indicating continued interest and research in this area. Advocates believe that, despite this setback, the path to FDA approval for psychedelic therapies remains promising.
Novartis’ IgAN Ambition Takes Shape With Expanded FDA Nod for FABHALTA
Novartis announced that the FDA has granted accelerated approval for FABHALTA® (iptacopan), a first-in-class complement inhibitor, for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. This approval is based on interim results from the Phase III APPLAUSE-IgAN study, which showed a significant 44% reduction in proteinuria at nine months compared to a 9% reduction in the placebo group.
“The heterogeneous and progressive nature of IgA nephropathy has made it challenging to effectively treat this disease. Thankfully, the treatment landscape is rapidly evolving,” said Professor Dana Rizk from the University of Alabama at Birmingham. “I am thrilled that this advancement is now available to help enable a targeted treatment approach for IgAN patients.”
IgAN is a rare disease where the immune system attacks the kidneys, causing inflammation and proteinuria, and affects around 25 people per million globally each year. Despite standard care, up to 50% of patients with persistent proteinuria progress to kidney failure within 10 to 20 years, often requiring dialysis or transplantation. FABHALTA’s favorable safety profile includes common adverse reactions such as upper respiratory tract infection, lipid disorder, and abdominal pain.
“Today’s approval of FABHALTA as a first-in-class medicine for IgA nephropathy is an important milestone in our journey to evolve rare renal disease care,” said Victor Bultó, President US, Novartis. The company is also developing two additional IgAN therapies: atrasentan and zigakibart, both in late-stage development. Bonnie Schneider, Director and Co-Founder of the IgAN Foundation, expressed hope, stating, “Today’s approval offers new hope for people living with IgA nephropathy as it represents a treatment innovation that provides us with a new way to fight this multifaceted disease.”
Servier Snags FDA Approval For Voranigo, The First Targeted Therapy for a Common Type of Brain Tumor
Servier announced that the FDA has approved VORANIGO®, an IDH1 and IDH2 inhibitor, for treating adults and pediatric patients aged 12 and older with Grade 2 astrocytoma or oligodendroglioma with susceptible IDH1 or IDH2 mutations following surgery. This approval marks a significant advancement in glioma treatment, providing patients with the convenience of a once-daily pill to manage their disease.
“Today’s approval of VORANIGO is an enormous leap forward in cancer care, and a defining moment for people living with Grade 2 IDH-mutant glioma,” said Arjun H. Prasad, Chief Commercial Officer, Servier Pharmaceuticals. “VORANIGO offers patients unprecedented improvement in progression-free survival.”
Gliomas, particularly those with IDH mutations, are common malignant brain tumors in adults under 50. VORANIGO works by inhibiting mutant IDH1 and IDH2 enzymes, helping control the disease. The Phase III INDIGO clinical trial demonstrated that VORANIGO significantly extended progression-free survival compared to placebo and was well-tolerated, with common adverse reactions including fatigue, COVID-19, musculoskeletal pain, diarrhea, and seizure.
“The FDA approval of VORANIGO marks a monumental breakthrough in glioma treatment, offering renewed hope for patients and their families,” said Ralph DeVitto, President & CEO of the American Brain Tumor Association. David K. Lee, CEO of Servier Pharmaceuticals, added, “VORANIGO can offer patients and their families hope for the future.”
