PharmaNewsIntelligence’s Post

This week, a panel of independent advisors to the Food and Drug Administration (FDA) recommended approval of Eli Lilly and Company's new Alzheimer’s drug, donanemab. This recommendation paves the way for #donanemab to receive FDA approval in the U.S. later this year, marking a significant milestone in the battle against a disease that affects millions of individuals and their families worldwide. The panel’s recommendation is based on compelling data from Eli Lilly’s clinical trial program in 2023. In the pivotal Phase 3 study, donanemab demonstrated a significant slowing of cognitive and functional decline in patients with early symptomatic Alzheimer’s disease. Results showed that patients treated with donanemab experienced a substantial reduction in the progression of the disease compared to those receiving placebo. What does this mean for patients? Per Joel Salinas, MD, MBA, MSc, FAAN, “This recommendation means that an external panel believes that donanemab has shown promising results in slowing cognitive decline in early symptomatic stages of #Alzheimers. Now, the remaining step is for the FDA to give its review.” Read more about donanemab in our blog post 👉 https://lnkd.in/eCr9CVx8

Part I: Rare Disease Drug Development: What patients and Advocates need to know. by National Organization for Rare Disorders FDA Foundation Fighting Blindness #raredisease #LCA10 #orphandrugs https://lnkd.in/dty4c4_Y

FDA doubles down on patient engagement to support rare disease research For Rare Disease Day, several panels organised by the FDA discussed the importance overcoming accessibility barriers during drug development. - https://lnkd.in/dz5DY4xt #rarediseaseday #raredisease #fda #clinicaltrials #drugdevelopment #patientengagement

Merck shares traded more than 4% higher after it won US Food and Drug Administration (FDA) approval for its lung disorder drug sotatercept, brand name WINREVAIR.  After being granted breakthrough therapy designation by the agency, the drug is now the first FDA-approved activin signalling inhibitor therapy for pulmonary arterial hypertension (PAH), a rare, progressive and life-threatening disease where the blood vessels in an individual’s lungs thicken and narrow and cause strain on the heart.   The approval is based on results from the Phase 3 STELLAR study which showed WINREVAIR in combination with background therapy significantly improved participants’ exercise capacity when compared with background therapy alone. More at #Proactive #ProactiveInvestors #NYSE #ETR #MRK #6MK http://ow.ly/EU6T105nv3E

We're excited to share that our collaborative project on Fabry disease(LA-GLA, code name: HM14521/GC1134A) with Hanmi Pharmaceutical, has just earned Orphan Drug Designation(ODD) from the FDA! Living with Fabry disease means enduring the inconvenience of bi-weekly, hours-long IV treatments at the hospital. But here comes LA-GLA, a game-changer! It's the first-ever treatment to offer a once-a-month subcutaneous injection, breaking new ground beyond the limitations of existing therapies. LA-GLA is proving superior to current treatments, with significant improvements noted in kidney function, vascular health, and peripheral nerve disorders. This February, two pivotal studies showcasing its benefits were presented at the WORLD Symposium 2024 in San Diego. Buoyed by these promising results, we are gearing up to file an IND application this year. *Fabry disease is a genetic disorder linked to the X chromosome, caused by deficient levels of the enzyme alpha-galactosidase A. This deficiency leads to cellular toxicity and inflammation, progressively damaging various organs and potentially escalating to a severe, life-threatening condition. https://lnkd.in/evM9FCGT #Fabry #LAGLA #ODD #FDA #Innovation #Partnering #Collaboration

At the World Orphan Drug Congress, Michael Fusakio and Samantha Z. held a fireside chat, ‘Rare but Not Forgotten: Challenges and Opportunities for Rare Disease Drugs,’ and talked through the challenges and implications in drug development for #rarediseases. They shared their own stories so that others could walk away with practical suggestions because developing therapies for rare diseases certainly has its own unique set of development and regulatory hurdles. But opportunities await! Here's a look into their Q&A. For questions about your clinical development plan and regulatory strategy, we’re ready when you are. https://hubs.la/Q02vYNF50 #WODC #RareDisease #RareDiseaseAwareness #ClinicalDevelopment #ClinicalTrials

🚨 Important Update in Medicines Development: The European Medicines Agency recommends against renewing the marketing authorization for Translarna, a drug for Duchenne muscular dystrophy, following a reassessment and new study findings that questioned its effectiveness. Learn more about conditional approvals and the challenges in ensuring timely access to therapies for rare diseases. #MedicineDevelopment #HealthcareNews #MedicalAffairs #GMDPacademy #EMA #translarna

The road to success is rarely straightforward, but today we celebrate a significant milestone in the fight against Alzheimer's. A #donanemab has shown promising effectiveness, bringing hope to millions of patients and their families. This breakthrough is a testament to the relentless dedication of researchers and medical professionals worldwide. Let's continue to support and drive innovation in medical research, for a future where Alzheimer's is a challenge we have conquered. #AlzheimersResearch #MedicalInnovation #HopeForTheFuture

Top Story Subcutaneous Leqembi filing delayed after FDA asks Eisai for more data Elizabeth S. Eaton PUBLISHED: APRIL 01, 2024 Ref: PR Newswire A much-anticipated subcutaneous (SC) version of Eisai and Biogen’s Alzheimer’s disease treatment is facing a setback after the FDA said the fast-track status held by the approved intravenous Leqembi (lecanemab-irmb) does not carry over to the new formulation, which will require its own submission – and push back a regulatory filing that was expected in March. The delay comes as the partners struggle to gain greater global traction for the anti-amyloid antibody. Last month, Eisai disclosed that less than 4000 patients have received Leqembi – far short of its 10,000-person goal by the end of fiscal year 2023. Meanwhile, a potential approval in the EU was pushed back after the European Medicines Agency (EMA) said it would need to hold another expert meeting after deciding to annul the advice obtained from a previous panel of independent experts due to “procedural reasons.”  The more convenient SC dosing is expected to boost use of the drug by eliminating the need for multiple hospital stays required for lengthy infusions. In a recently fielded FirstWord poll, 62% of responding neurologists indicated that they view SC Leqembi as more than somewhat superior to the currently marketed version. Additionally, an overwhelming majority predicted the formulation would help provide greater access to the drug for those in rural areas of the country, which are typically medically underserved.

Continuing on from my post yesterday, today's post will focus on Alzheimer's Disease (AD). AD is the most common type of dementia and is pathologically characterized by amyloid plaques and tau tangles and behaviourally characterized by a gradual decline in memory. The treatment of AD has evolved significantly over the years, with various drugs targeting different aspects of the disease's progression. Here's a brief history: 💊 Cholinesterase Inhibitors (1993 onwards): The first class of drugs approved for Alzheimer's treatment were cholinesterase inhibitors. These drugs, including tacrine (Cognex), donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), work by increasing levels of acetylcholine, a neurotransmitter involved in memory and learning.Tacrine was the first drug approved by the FDA in 1993, but its use declined due to safety concerns and the development of more effective alternatives. ⚕ NMDA Receptor Antagonist (2003): Memantine (Namenda) was approved by the FDA in 2003 as the first drug in a new class of Alzheimer's medications. It works by modulating the activity of glutamate, another neurotransmitter involved in learning and memory, and is believed to protect brain cells from excessive stimulation. 💊 ⚕ Combination Therapy: In some cases, healthcare providers may prescribe a combination of cholinesterase inhibitors and memantine to provide synergistic effects in managing symptoms of Alzheimer's disease. 🏥 Research and Clinical Trials: Numerous other drugs have been studied in clinical trials for Alzheimer's treatment, targeting various aspects of the disease such as amyloid plaque accumulation, tau protein tangles, inflammation, and oxidative stress. However, many of these drugs have failed to show significant efficacy in late-stage trials, leading to disappointment in the search for a definitive cure or disease-modifying treatment. 👨🔬 Biogens Aducanumab (2021): In June 2021, the FDA granted accelerated approval to aducanumab (brand name Aduhelm) for the treatment of AD. Aducanumab targets amyloid beta, a protein that forms plaques in the brains of people with Alzheimer's disease. The approval was controversial due to conflicting evidence regarding the drug's efficacy, with some clinical trials showing modest benefits while others did not demonstrate significant improvement in cognitive function. The New York Times article below, focusing on Eli Lilly and Companys latest therapy to try and treat AD shows the complexity of getting these drugs over the line. The FDA has postponed its decision on the drug, donanemab, opting for further review by an independent panel of experts, surprising both experts and the drug's manufacturer. The delay reflects the FDA's cautious approach, particularly in evaluating unique trial designs and addressing concerns about treatment cessation and the role of tau proteins. https://lnkd.in/e3uE8Ptj