From development of the first treatment for Niemann-Pick disease to approval of a medication that reduces the risk of cardiovascular death and heart failure hospitalization, learn about the most impactful changes at each stage of the drug pipeline in this edition of Perspective on the Rx Pipeline.

Perspective on the Rx Pipeline

Clinical Pipeline

Within the extensive pipeline of drugs preparing for market launch, these are the two most notable drugs plan sponsors should be aware of:

• Arimoclomol would be the first FDA-approved treatment for the rare and progressive genetic disorder Niemann-Pick disease type C, which causes the build-up of fatty substances inside cells and leads to death at an early age. Current treatment options only provide symptom management. 
• KD025, a selective inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), would be the first FDA-approved oral option of this mechanism of action. It is being developed for the treatment of graft versus host disease, a common complication of allogenic hematopoietic cell transplant, and has additional indications in the pipeline.  

Drug Approvals

Eight drugs recently approved by the FDA include:

• Gemtesa® (vibegron), the first FDA-approved treatment for overactive bladder since 2012.  
• Imcivree™ (setmelanotide), a weight management treatment that is limited to those with specific genetic obesity disorders.
• Klisyri® (tirbanibulin), a new mechanism of action for the treatment of actinic keratosis that is self administered.
• Orgovyx® (relugolix), an oral androgen deprivation therapy (ADT) option for the treatment of prostate cancer.
• Orladeyo™ (berotralstat), the first oral preventive therapy for hereditary angioedema (HAE).
• Oxlumo™ (lumasiran), a treatment option for the ultra-rare genetic disease primary hyperoxaluria type 1 (PH1).
• Verquvo® (vericiguat), an add-on therapy for heart failure management with a unique mechanism of action that reduces the risk of cardiovascular death and heart failure hospitalization.
• Zokinvy™ (ionafarnib), the first drug approved for the treatment of progeria, a disease that causes premature aging.

New Indications

Drugs that have gained FDA approval for the treatment of additional diseases and/or conditions and have potential to be impactful include:

• Benlysta® (belimumab), expanded to treat adult patients with active lupus nephritis.
• Gavreto™ (pralsetinib), expanded to treat patients 12 years of age and older with RET-mutant medullary and RET fusion-positive thyroid cancer.
• Hetlioz® (tasimelteon) received an additional indication for the treatment of nighttime sleep disturbances in Smith-Magenis Syndrome patients.
• Wakix® (pitolisant) gained a cataplexy indication in the treatment of adult patients with narcolepsy.
• Xofluza® (baloxavir marboxil) received the updated indication of post-exposure prophylaxis of influenza in patients 12 years of age and older following contact with an individual who has influenza.
• Xolair® (omalizumab), granted the indication of add-on maintenance treatment of nasal polyps in adults. 

Generic Approvals

There are several new generic product offerings to the marketplace, including drugs for the treatment of osteoarthritis pain, prostate cancer and bipolar disorder. A review of generic launches, including biosimilars, is provided in the full report.

Download the full report for more information, including important safety updates and drug shortages and discontinuations.