Regulatory Updates

Clarivate analysts have identified the treatments they forecast could deliver annual sales of more than $1 billion within five years. These include therapies for rare diseases, as well as HIV, Parkinson’s disease, Crohn’s disease, alopecia, and cancer.

The agency has assigned a Prescription Drug User Fee Act target action date of May 10, 2023, and plans to hold an advisory Committee

The supplemental BLA is based on data from a confirmatory phase 3 trial. The submission was made the same day Eisai/Biogen received accelerated approval.

Lecanemab, now with the brand name Leqembi, will launch with a wholesale acquisition cost of $26,500 a year.

Genentech’s glofitamab is a bispecific antibody being reviewed to treat adults with large B-cell lymphoma. The FDA is expected to make a decision on approval by July 1, 2023.

Rozanolixizumab is a monoclonal antibody that addresses a different driver of generalized myasthenia gravis. An FDA decision is expected in the second quarter of 2023.

Nirsevimab is a single-dose, long-acting antibody to protect infants from respiratory syncytial virus (RSV) lower respiratory tract infections. The FDA action date is in the third quarter of 2023.

CT-P13 SC is a subcutaneous formulation of Celltrion’s Inflectra, a biosimilar of Remicade. The BLA seeks approval to treat patients with inflammatory bowel disease.

Wegovy is a once-weekly prescription medication for obesity that has faced supply issues. Its manufacturer, Novo Nordisk, is increasing production capacity in 2023.

If approved, CSF-1 would be the second therapy to treat patients with presbyopia, or age-related blurry vision.

Olpruva is a new formulation of sodium phenylbutyrate to treat patients with urea cycle disorders, a genetic condition that causes ammonia to build up in the blood.

Ublituximab, now with brand name Briumvi, is the first anti-CD20 monoclonal antibody approved for patients with multiple sclerosis that can be administered in a one-hour infusion twice-a-year.

Palovarotene is a therapy for an ultra-rare disease that transforms the body’s soft tissue into bone. The FDA has requested additional information from clinical trials.

A decision by the FDA is expected by April 28, 2023, and, if approved, Harm Reduction Therapeutics will launch RiVive in early 2024.

Among the top-read FDA approval stories include the fast-acting Auvelity to treat major depressive disorder; an extended use for Mirena IUD; the first generic of Symbicort for asthma; Camzyos, a first-in-class treatment for obstructive hypertrophic cardiomyopathy; and Imbruvica’s expanded indication for children with chronic graft versus host disease.

Lunsumio is Genentech’s first approved T-cell engaging bispecific antibody and targets CD20 on the surface of B cells and CD3 on the surface of T cells.

Lenacapavir, now with the brand name Sunlenca, is a twice a year therapy for adult patients with multi-drug resistant HIV. It has a list price of $42,250 for the first year.

The combination includes an antibody-drug conjugate with an immunotherapy to treat patients with advanced bladder cancer. The PDUFA action date is April 21, 2023.

Actemra is the first FDA-approved monoclonal antibody to treat hospitalized adults with COVID-19. The EUA for use of Actemra in children remains in place.

Tymlos is already available to treat postmenopausal women with osteoporosis.

This is the fourth indication for Vraylar, a dopamine and serotonin partial agonist that can now be used as an adjunctive therapy in patients with major depressive disorder.

Cytalux, approved in November 2021 for identifying ovarian cancer lesions, is now approved to spot lung cancer during surgery.

If approved, Linzess would be the first prescription therapy for functional constipation in children and adolescents 6 to 17 years of age.

Adstiladrin offers an alternative to bladder removal surgery for patients who are at high risk of recurrence and metastatic cancer.

Iyuzeh is a preservative-free formulation of latanoprost and doesn’t need to be stored at refrigerated temperatures. It will be available in the second half of 2023.

HyBryte is a photodynamic therapy. The active ingredient, synthetic hypericin, is topically applied to skin lesions that is taken up by the malignant T-cells and activated by visible light.

This is the eighth biosimilar to reference the blockbuster rheumatoid arthritis drug Humira that will launch in 2023.

The agency has extended the review time by three months for Lynparza in combination with abiraterone and prednisone or prednisolone.

Building on the success of the companies’ COVID-19 vaccines, Pfizer and BioNTech have begun a phase 1 trial of a combined flu-COVID-19 vaccine.

The FDA issued an accelerated approval for Mirati’s Krazati for patients with KRAS-mutated non-small cell lung cancer. The agency also approved a companion diagnostic to identify eligible patients.