From development of the first oral preventive medication for hereditary angioedema to approval of the first oral disease-modifying therapy for spinal muscular atrophy, 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 three most notable drugs plan sponsors should be aware of:

• Inclisiran, a small interfering RNA molecule (siRNA) being developed as an adjunct to diet to be used in conjunction with statin therapy for additional lipid lowering. This medication has a similar mechanism of action to currently available products, but with less frequent maintenance injections.
• Orladeyo, a kallikrein inhibitor in development for prophylaxis treatment of hereditary angioedema (HAE) that, if approved, would offer the first oral preventative treatment option for this rare life-threatening condition.  
• RM-493, a melanocortin-4 (MC4R) agonist being developed as a very specific weight loss treatment for obesity due to rare genetic disorders that was recently granted Breakthrough Therapy Designation by the FDA.    

Drug Approvals

Seven drugs recently approved by the FDA include:

• Evrysdi™ (risdiplam), the first oral disease-modifying therapy for the treatment of spinal muscular atrophy (SMA).  
• Kesimpta® (ofatumumab), an anti-CD20 monoclonal antibody for the treatment of clinically isolated syndrome, relapsing-remitting disease and active secondary progressive multiple sclerosis that can be self-administered.
• Mycapssa® (octreotide), for long-term maintenance treatment of acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.
• Veklury® (remdesivir), the first FDA-approved treatment for COVID-19.
• Viltepso® (viltolarsen), another intravenous medication for the treatment of Duchenne muscular dystrophy (DMD) in patients where the DMD gene is exon 53 skipping.
• Winlevi® (clascoterone), the first FDA-approved new mechanism of action for the treatment of acne in over three decades.
• Xywav™ (calcium oxybate, magnesium oxybate, potassium oxybate, sodium oxybate), an approved treatment for cataplexy or excessive daytime sleepiness associated with narcolepsy that contains 92% less sodium than currently available treatments.

New Indications

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

• Epidiolex® (cannabidiol), now indicated to treat seizures associated with tuberous sclerosis.
• Kalydeco® (ivacaftor), expanded to the youngest FDA-approved indication of cystic fibrosis, at four months of age and older.
• Nucala® (mepolizumab), now indicated for adult and pediatric patients 12 years of age and older with hypereosinophilic syndrome (HES).
• Spravato® (esketamine), granted a new indication to treat depressive symptoms in adults with major depressive disorder (MDD) with suicidal thoughts or actions.
• Stelara® (ustekinumab), received the additional indication of moderate to severe plaque psoriasis in pediatric patients as young as six years of age.

Generic Approvals

There are several new generic product offerings to the marketplace, including drugs for the treatment of HIV, diabetes and schizophrenia. 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.