This week, the FDA has an exciting agenda lined up, with a target action date and an advisory committee meeting that could be a game-changer for the neurodegenerative disease space. On top of that, it’s prepping for yet another advisory committee meeting that could prove to be a pivotal moment for the field. All eyes are on the FDA this week!
Apellis Seeks Approval for Empaveli Injector
On March 15, the future of Apellis Pharmaceuticals’ groundbreaking new treatment for paroxysmal nocturnal hemoglobinuria (PNH) will be decided by the FDA. The sNDA for the injectable Empaveli (pegcetacoplan) will be evaluated, potentially offering a long-awaited solution to those suffering from PNH.
Say goodbye to needles and syringes! Empaveli now offers patients a custom drug delivery system that allows them to self-administer subcutaneous infusions quickly and easily. Attached to the body, the injector provides a convenient and pain-free way to get the medication you need. Enjoy the freedom and ease of this revolutionary drug delivery system today!
In May 2021, the FDA approved Empaveli, a revolutionary complement inhibitor, for treating PNH – a rare disorder caused by an uncontrolled activation of the immune system’s complement cascade. This novel treatment works by targeting C3, the central cascade protein, to halt the destruction of red blood cells and provide relief for those living with PNH.
Empaveli is a revolutionary PNH therapeutic that targets C3, a critical component of the complement system. Unlike other treatments, which only target the terminal complement protein CD5, Empaveli works by delivering a powerful 1,080-mg dose of the drug twice a week via a simple subcutaneous infusion. Patients can easily administer the treatment using a commercially available pump, providing a convenient and effective way to manage the disease.
Empaveli carries a stark warning for meningococcal infections – they can quickly become fatal if not detected and treated promptly. Moreover, those taking Empaveli may be at greater risk of serious infections, particularly from encapsulated bacteria. It is essential to be vigilant and seek medical attention if any symptoms of infection arise.
Empaveli is a powerful tool, but comes with inherent risks. That’s why access is limited to a special program with a Risk Evaluation and Mitigation Strategy, ensuring users stay safe while enjoying its benefits.
Adcomm Convenes for Pfizer’s Paxlovid
The FDA is set to make a pivotal decision on March 16, when its Antimicrobial Drugs Advisory Committee will meet to assess whether Pfizer’s COVID-19 drug, Paxlovid, should be granted full approval. This drug, a co-packaged tablet containing nirmatrelvir and ritonavir, could prove to be a game-changer in the fight against the virus.
Pfizer is set to present Paxlovid as a potential treatment for mild-to-moderate COVID-19 in adult patients at risk of developing severe symptoms, hospitalization or death. All the important information regarding the meeting will be made available to the public no later than two days before the panel convenes. This could be a major step forward in the fight against the virus and its potentially deadly consequences.
Paxlovid is an innovative antiviral medication that combines the power of nirmatrelvir and ritonavir to fight COVID-19. By slowing down the liver’s detoxification activity, Paxlovid can stay active in the body for a longer period of time, making it an effective treatment for mild-to-moderate cases of the virus. It has received emergency authorization from the FDA and is now available to help combat the pandemic.
In June 2022, Pfizer submitted a New Drug Application for regular approval of Paxlovid – a potential breakthrough treatment for both vaccinated and unvaccinated individuals. With the EPIC-HR study providing data showing an 86% decrease in hospitalizations and death from all causes relative to placebo, the company is optimistic that Paxlovid could revolutionize the treatment of Covid-19.
In August 2022, the FDA issued a request to Pfizer to study the potential benefits of an extra course of its coronavirus drug for patients who had developed rebound infections. This was a significant step forward in the ongoing fight against the virus, as the drug could prove to be a powerful ally in tackling the virus’s recurrence in vulnerable populations.
On the Horizon: Biogen/Ionis Adcomm for Tofersen
The FDA is preparing for an upcoming advisory committee meeting that could be a major milestone in the fight against ALS and other neurodegenerative diseases. With the potential to revolutionize treatments, this meeting could be a crucial step towards bringing hope to those affected by these conditions.
On March 22, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee will be meeting to review Biogen’s New Drug Application for tofersen, a potential treatment for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). This is a significant milestone in the development of therapies for this debilitating neurodegenerative disease.
In 2018, Biogen made a groundbreaking agreement with Ionis Pharmaceuticals to license their revolutionary new discovery, Tofersen. This has been a major step forward in the field of medicine, and is sure to have a great impact in the years to come.
On March 22, Biogen’s New Drug Application for tofersen, a potential therapy for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), will be reviewed by the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee. If approved, this could be a major breakthrough in the fight against this devastating neurodegenerative disease, providing hope to those affected by ALS.
In 2018, Biogen struck a deal with Ionis Pharmaceuticals to license Tofersen, an innovative discovery that has the potential to revolutionize treatments for neurological diseases. This groundbreaking agreement promises to bring new hope to those affected by debilitating illnesses.
Tofersen is an innovative therapy being studied to treat ALS caused by mutations in the SOD1 gene. This cutting-edge antisense oligonucleotide is specifically designed to target the mRNA of the faulty SOD1 protein and reduce its production, offering a potential solution for those living with this devastating disease.
Biogen is set to make a major impact on the neurodegenerative diseases landscape, as the company has submitted a New Drug Application (NDA) for tofersen in July 2022. The NDA largely focuses on tofersen’s ability to lower neurofilament levels, a biomarker that is increasingly gaining traction in the field. This drug application will be a key moment for the medical community and the adcomm will be a pivotal test of its efficacy.
The FDA initially set a target action date for tofersen of January 25, 2023, but after Biogen provided additional data in October 2022, the FDA moved the goal date back to April 25, 2023. This three month delay provides Biogen with more time to further develop their drug and ensure its efficacy before it is approved for use.