Specialty drugs continue to be one of the main categories in the FDA\u2019s pipeline.<\/p>\n\n\n\n
According to a session at Asembia\u2019s AXS23 summit, they influence the new FDA approvals.<\/span><\/p>\n\n\n\n In recent years, the pharmaceutical industry has witnessed a surge in developing and approving groundbreaking medications. <\/p>\n\n\n\n Ray Tancredi, an industry expert at Walgreens, shared insights into the growing pipeline of specialty drugs poised to make a significant impact.<\/span> <\/p>\n\n\n\n With a focus on oncology, rare diseases, and cell and gene therapies, Tancredi shed light on the potential of these novel treatments. <\/p>\n\n\n\n In this blog post, we will explore some of the notable drugs that have recently been approved or are expected to be approved soon.<\/p>\n\n\n\n According to Tancredi, 2023 has started off on a promising note, with 14 new drug approvals<\/span><\/em><\/strong> <\/em>already in place. <\/p>\n\n\n\n Out of these, 12<\/span><\/strong><\/a> <\/span>are specialty medications.<\/p>\n\n\n\n Additionally, 30 specialty drugs<\/span><\/strong> are anticipated to be approved by the end of the year.<\/p>\n\n\n\n Let us take a closer look at some of the remarkable advancements within the realm of rare diseases and other therapeutic areas.<\/p>\n\n\n\n 1. Sparsentan<\/span><\/strong><\/p>\n\n\n\n This innovative drug was approved by the FDA in February.<\/p>\n\n\n\n It is the first single-molecule dual endothelin angiotensin receptor antagonist to receive approval for patients with immunoglobulin A nephropathy (IgAN). <\/p>\n\n\n\n Sparsentan<\/span><\/a> aims to reduce proteinuria and is currently undergoing phase 3 trials for focal segmental glomerulosclerosis.<\/p>\n\n\n\n 2. Leniolisib<\/span><\/strong><\/p>\n\n\n\n In March, leniolisib<\/span><\/a> became the first approved treatment for activated phosphoinositide 3-kinase delta syndrome. <\/p>\n\n\n\n Clinical data demonstrated its ability to reduce lymph node size and improve naive B cell counts, offering hope for patients with this condition.<\/p>\n\n\n\n 3. Tofersen<\/strong><\/span><\/p>\n\n\n\n On April 25, the FDA approved tofersen<\/span><\/a> <\/span>for amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. <\/p>\n\n\n\n This targeted therapy is expected to benefit a subset of ALS patients with a genetic component, addressing an unmet need.<\/p>\n\n\n\n 4. Nirogacestat<\/span><\/strong><\/p>\n\n\n\n Investigated as a treatment for desmoid tumors, nirogacestat<\/span><\/a> has shown significant benefits in terms of progression-free survival, symptom relief, and quality of life improvements during phase 3 trials. <\/p>\n\n\n\n The drug has received Fast Track Designation and Orphan Drug Status and may be launched by the end of this year.<\/p>\n\n\n\n 5. Fitusiran<\/span><\/strong><\/p>\n\n\n\n Antithrombin RNA interference therapeutic fitusiran<\/span><\/a> has demonstrated significant improvements in bleed protection for patients with hemophilia A and B during phase 3 studies. <\/p>\n\n\n\n Pending approval, this promising therapy could be available as early as December.<\/p>\n\n\n\n 6. Sotatercept<\/span><\/strong><\/p>\n\n\n\n Designed to treat pulmonary arterial hypertension (PAH), sotatercept<\/span><\/a> has shown promise in improving patients’ exercise capacity, a critical factor in managing this life-threatening condition. <\/p>\n\n\n\n The launch of the sotatercept could potentially address the pressing need for better treatments for PAH patients.<\/p>\n\n\n\n 7. Nipocalimab<\/strong><\/span><\/p>\n\n\n\n Being investigated for hemolytic disease of the fetus and newborn (HDFN), nipocalimab<\/span><\/a> has displayed promising results in a phase 2 trial, with a high rate of successful live births among pregnant patients at high risk for severe HDFN.<\/p>\n\n\n\nThe Potential FDA Drug Approvals<\/strong><\/h2>\n\n\n\n