FDA Update for Week of May 23, 2022

FDA approves Amneal’s biosimilar of Neulasta.

The FDA has approved Amneal Pharmaceuticals’ biologics license application for Fylnetra (pegfilgrastim-pbbk), a biosimilar referencing Amgen’s Neulasta (pegfilgratim). Fylnetra was developed in collaboration with Kashiv Biosciences. It is used to decrease the risk of infection after chemotherapy.

Four other biosimilars have been approved by the FDA: Fulphila, co-developed by Biocon Biologics and Mylan; Nyvepria, developed by Pfizer; Udenyca, developed by Coherus BioSciences; and Ziextenzo, developed by Sandoz, a division of Novartis.

This marks the third biosimilar approval Amneal received this year for products used in oncology. Earlier this year, the FDA approved Releuko (filgrastim-ayow), a filgrastim biosimilar referencing Neupogen, and Alymsys (bevacizumab-maly), a bevacizumab biosimilar referencing Avastin. Amneal expects to launch these three products over the second half of 2022, along with a full patient support program.

FDA approves first non-steroid cream for psoriasis.

The FDA has approved Vtama (tapinarof) for the topical treatment of plaque psoriasis in adults. This is the first steroid-free topical medication in its class. It is approved for mild, moderate, and severe psoriasis with no restrictions on duration of use. Vtama was developed by Dermavant Sciences, a subsidiary of Roivant Sciences.

Vtama is an aryl hydrocarbon receptor agonist and modifies the expression of interleukin-17 and the skin barrier proteins filaggrin and loricrin, which are involved in psoriasis. About 8 million Americans and 125 million people worldwide are affected by psoriasis, which is a complex autoimmune disease. Plaque psoriasis is the most common form and affects about 80% to 90% of people with psoriasis.

FDA approves Tibsovo combination for older patients with leukemia.

The FDA has approved Servier Pharmaceuticals’ Tibsovo (ivosidenib) in combination with azacitidine for the treatment of patients with newly diagnosed IDH1-mutated acute myeloid leukemia (AML). It is indicated for adults 75 years or older or those who are ineligible for intensive induction chemotherapy.

Tibsovo is the first therapy targeting cancer metabolism approved in combination with azacitidine for patients with newly diagnosed IDH1-mutated AML.

AML is a difficult-to-treat cancer of the blood and bone marrow and is one of the most common types of leukemia in adults with about 20,000 new cases estimated in the United States each year. The IDH1 gene provides instructions for making the enzyme isocitrate dehydrogenase 1, which produces a molecule necessary for cellular processes. IDH1 mutations are present in about 6% to 10% of AML cases.

FDA approves new formulation of Tyvaso.

FDA has approved United Therapeutics’ Tyvaso DPI (treprostinil) inhalation powder to treat patients with pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease. Tyvaso DPI is a new formulation and inhalation device for inhaled treprostinil and is the only dry powder inhaler approved by the FDA for these indications.

Pulmonary arterial hypertension is life-threatening high blood pressure in the arteries of the lungs, affecting the ability of the heart and lungs to work properly. It affects an estimated 45,000 patients in the United States. Interstitial lung disease is a group of conditions in which marked scarring occurs within the lungs. It is often complicated by pulmonary hypertension, which is estimated to affect at least 15% of patients with early-stage ILD.

Tyvaso Inhalation Solution and Tyvaso DPI are the only therapies approved by the FDA to treat pulmonary hypertension associated with interstitial lung disease.

FDA issues second CRL for poxvirus treatment.

The FDA has issued a complete response letter (CRL) for Verrica Pharmaceuticals’ new drug application (NDA) for VP-102 for the treatment of molluscum contagiosum, an infection caused by poxvirus. This infection is usually a mild skin disease characterized by lesions that may appear anywhere on the body, according to the CDC. But there is currently no approved treatments for this infection, which affects about 6 million people worldwide, mostly children.

VP-102 is a proprietary drug-device combination product that contains a formulation of cantharidin delivered via a single-use applicator that allows for topical dosing and targeted administration. If approved, it would be the first product approved to treat molluscum contagiosum.

The FDA indicated in the CRL that there were deficiencies identified at a general reinspection of Sterling Pharmaceuticals, the contract manufacturing organization that manufactures Verrica’s bulk solution drug product.

Sterling advised Verrica on May 20, 2022, that it received notice from the called Official Action Indicated (OAI). This was the result of a week-long reinspection of Sterling conducted by FDA in February 2022. The reinspection was conducted about 90 days after Sterling was originally classified by the agency as Voluntary Action Indicated (VAI) on November 17, 2021.

Verrica indicated in a press release no other deficiencies indicated and that the deficiencies weren’t specific to VP-102.

FDA accepts NDA for omaveloxolone for Friedreich’s ataxia.

The FDA has accepted for filing and granted priority review of the new drug application for Reata Pharmaceuticals’ omaveloxolone for the treatment of patients with Friedreich’s ataxia. Friedreich’s ataxia is a rare, genetic, degenerative neuromuscular disorder. If approved, the company anticipates omaveloxolone will be available in early 2023.

Patients with Friedreich’s ataxia typically experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue that commonly results in motor incapacitation with patients requiring a wheelchair in their 20s. It impacts about 5,000 children and adults in the United States and 22,000 individuals globally. Currently, there are no approved therapies for the treatment of Friedreich’s ataxia.

Omaveloxolone is an investigational, oral, once-daily activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling.

FDA accepts NDA for nasal migraine treatment.

The FDA has accepted for the new drug application (NDA) of Biohaven’s zavegepant nasal spray for the acute treatment of migraine in adults. The Prescription Drug User Fee Act (PDUFA) goal date for completion for the first quarter of 2023.

Zavegepant a small molecule calcitonin gene-related peptide (CGRP) receptor antagonist in an intranasal formulation. If approved it would be the only receptor antagonist in an intranasal formulation, providing pain relief in 15 minutes that lasts through 48 hours after a single dose.
Zavegepant is a third-generation small molecule CGRP receptor antagonist from Biohaven's migraine platform and the only CGRP receptor antagonist in clinical development with both intranasal and oral formulations.

Biohaven also markets Nurtec and Nurtec ODT (rimegepant), which was approved by the FDA first approved in February 2020 for the acute treatment of migraine in adults.

In May 2022, Pfizer agreed to acquire Biohaven for about $11.6 billion in cash.

FDA accepts BLA for mirvetuximab soravtansine for ovarian cancer.

The FDA has accepted the biologics license application (BLA) for ImmunoGen’s mirvetuximab soravtansine as a monotherapy for patients with ovarian cancer. The application has been granted priority review designation and FDA has set a Prescription Drug User Fee Act (PDUFA) action date of November 28, 2022.

Mirvetuximab soravtansine (IMGN853) is a first-in-class antibody-drug conjugate comprising a folate receptor alpha-binding antibody, cleavable linker, and a potent tubulin-targeting agent, to kill the targeted cancer cells. It is intended for patients with folate receptor alpha (FRα)-high platinum-resistant ovarian cancer who have been previously treated with one to three prior systemic treatments.

ImmunoGen continues to enroll patients in the confirmatory MIRASOL trial. The company expects to announce top-line data from this study in early 2023.