Monthly Archives: September 2023

Biogen Inc. announced that the U.S. Food and Drug Administration has approved Tofidence (tocilizumab-bavi) intravenous formulation, a biosimilar monoclonal antibody referencing Actemra. The Tofidence intravenous formulation is approved for the treatment of moderately to severely active rheumatoid arthritis, polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis.

Tofidence is the first tocilizumab biosimilar approved in the United States. Biosimilars are biologic products that have been demonstrated to have equivalent efficacy and comparable safety as the approved reference product, with the advantage that they may offer cost savings and promote expanded and sustainable access to therapies.

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FDA Approves Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) for the Treatment of Late-Onset Pompe Disease

Amicus Therapeutics announced that the U.S. Food and Drug Administration has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

Pombiliti + Opfolda is a unique two-component therapy. Pombiliti is a recombinant human GAA enzyme (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), designed for increased uptake into muscle cells. Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood. Amicus Therapeutics will launch Pombiliti with Opfolda immediately in the U.S.

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FDA Approves Exxua (gepirone) for the Treatment of Major Depressive Disorder in Adults

Fabre-Kramer Pharmaceuticals Inc. (Fabre-Kramer) announced that the U.S. Food and Drug Administration has approved Exxua (gepirone hydrochloride extended-release tablets) for the treatment of major depressive disorder (MDD) in adults.

Exxua represents a new class of antidepressant; the first and only approved antidepressant with a novel mechanism of action that selectively targets the serotonin 1A receptor, a key regulator of mood and emotion. Exxua has been shown to effectively relieve depressive symptoms, and its approved labeling does not contain warnings or adverse reactions regarding causing sexual dysfunction or weight gain vs. placebo.

Exxua is expected to be available in pharmacies in early 2024. Exxua is not approved for use in pediatric patients.

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Scynexis, Inc. is conducting a voluntary nationwide recall of 2 lots of BREXAFEMME® (ibrexafungerp tablets) to the consumer level in the US market due to potential cross contamination with a non-antibacterial ß-lactam drug substance in the ibrexafungerp citrate used to manufacture the BREXAFEMME® tablets.

Risk Statement: The potential cross contamination with a non-antibacterial beta-lactam drug substance could lead to hypersensitivity reactions such as swelling, rash, urticaria and anaphylaxis, a potentially life-threatening adverse reaction. To date, SCYNEXIS has not received any reports of adverse events established to be due to the possible beta-lactam cross contamination.

The affected BREXAFEMME® lots include the following lots and expiration dates: LF21000008 (expiration date 11/2023) and LF22000051 (expiration date 11/2025). The recalled lots were distributed nationwide to wholesalers across the US, beginning in December 2022.

Scynexis is engaging with Sedgwick to manage the recall of the product down to the consumer level. Sedgwick will be notifying BREXAFEMME® distributors via a recall notification letter and will be arranging for the return of the recalled lot from distributors, retailers, and consumers.

Consumers with questions regarding this recall can contact Sedgwick at 1-877-551-7154. Office hours: Monday to Friday, 8:00 AM to 5:00 PM ET.

Consumers should contact their healthcare provider if they have experienced any problems that may be related to taking or using this drug product.

FDA Approves Ryzumvi (phentolamine) Ophthalmic Solution for the Treatment of Pharmacologically-Induced Mydriasis

Ocuphire Pharma, Inc., a clinical-stage ophthalmic biopharmaceutical company and Viatris Inc., announced that the U.S. Food and Drug Administration (FDA) has approved Ryzumvi™ (phentolamine ophthalmic solution) 0.75% for the treatment of pharmacologically-induced mydriasis produced by adrenergic agonists (e.g., phenylephrine) or parasympatholytic (e.g., tropicamide) agents.

In the U.S., an estimated 100 million comprehensive eye exams take place each year that involve pharmacologically-induced mydriasis (or dilation) of the pupils, which can last up 24 hours. Side effects of pharmacologically-induced mydriasis include sensitivity to light (photophobia) and blurred vision, which may make it difficult to read, work and drive. Ryzumvi is expected to be commercially available in the U.S. in the first half of 2024.

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FDA Approves Likmez (metronidazole) Oral Suspension for the Treatment of Parasitic and Anaerobic Bacterial Infections

Appili Therapeutics Inc., announced that its manufacturing and commercialization partner, Saptalis Pharmaceuticals, LLC received approval from the U.S. Food and Drug Administration for Metronidazole Oral Suspension 500mg/5mL (ATI-1501). ATI-1501. The FDA also approved Likmez™ as the brand name for ATI-1501.

Metronidazole is a widely used frontline oral treatment with over 10 million prescriptions written in the United States every year to help treat parasitic and anaerobic bacterial infections. The current tablet form of metronidazole is the only other approved oral form on the U.S. market, but its bitter taste and lack of appropriate dosage forms for patients with difficulty swallowing often presents treatment compliance challenges.

Likmez™ is the first and only FDA approved ready-made suspension of metronidazole for the treatment of antimicrobial infections that addresses the unmet need in patients with dysphagia and avoids risks associated with drug compounding, and discontinuation related anti-microbial resistance.

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The U.S. Food and Drug Administration has issued warning letters to eight companies for manufacturing or marketing unapproved ophthalmic drug products in violation of federal law. These warning letters are part of the agency’s ongoing effort to protect Americans from potentially harmful ophthalmic products.

Eye products addressed in the eight warning letters are illegally marketed to treat conditions such as conjunctivitis (“pink eye”), cataracts, glaucoma, and others. Some of the FDA warning letters also cite the companies involved for quality issues related to product sterility.

The FDA is particularly concerned that these illegally marketed, unapproved ophthalmic drug products pose a heightened risk of harm to users because drugs applied to the eyes bypass some of the body’s natural defenses. Some of these eye products are labeled to contain silver, which may be characterized as silver sulfate, silver sulphate or argentum. Long-term use of drugs containing silver can cause some areas of the skin and other body tissues, including in the eye, to permanently turn gray or blue-gray, which is called “argyria.” Additionally, unapproved drugs that claim to cure, treat, or prevent serious conditions may cause consumers to delay or stop medical treatments that have been found safe and effective through the FDA review process.

The agency issued warning letters to the following companies:

• Boiron Inc.
• CVS Health
• DR Vitamin Solutions
• Natural Ophthalmics, Inc.
• OcluMed LLC
• Similasan AG/Similasan USA
• TRP Company, Inc.
• Walgreens Boots Alliance, Inc.

Consumers currently using eye products included in these warning letters should speak to their health care professional. The FDA encourages consumers and health care professionals to report any adverse reaction to the agency’s MedWatch program.

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Revance Therapeutics, Inc. has announced that the United States (U.S.) Food and Drug Administration has approved the first therapeutic indication for Daxxify® (DaxibotulinumtoxinA-lanm) for injection for the treatment of cervical dystonia (CD) in adults. Daxxify® was previously approved by the FDA for the temporary improvement of glabellar lines (frown lines) in adults in September 2022 and is the first true innovation in neuromodulator product formulation in more than 30 years.

Currently, there are four other commercially available botulinum toxin (BoNT) products that have FDA-approved therapeutic indications, including CD. CD is a chronic, debilitating disease that affects an estimated 60,000 people in the United States. The condition is characterized by the involuntary contraction of neck muscles, which causes abnormal movements, pain, and awkward posture of the head and neck. Neuromodulators are considered the first line of treatment for this condition; Daxxify is a peptide-formulated neuromodulator.

Daxxify’s expanded approval for CD was based on data from the Phase 3 ASPEN clinical program (ASPEN-1 and ASPEN open-label study [OLS]), which included 382 patients with moderate to severe CD. In ASPEN-1, participants received a single low dose of Daxxify (125 units), a high dose of Daxxify (250 units), or placebo.

In ASPEN-OLS, symptoms continued to improve with successive Daxxify treatments at doses of up to 300 units, while adverse events remained low. Revance expects to secure a J-code for Daxxify by the end of 2023, with broad commercial availability expected in early 2024. The annual wholesale acquisition cost for Daxxify for the treatment of CD using the recommended dose range of 125-250 units four times per year is about $3,360–$5,040.

Daxxify will compete with AbbVie’s Botox (onabotulinumtoxinA), Ipsen’s Dysport (abobotulinumtoxinA), Merz Pharmaceuticals’ Xeomin (incobotulinumtoxinA), and Solstice Neurosciences’ Myobloc (rimabotulinumtoxinB), all of which are FDA-approved for the treatment of CD.

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Three poly (ADP-ribose) polymerase inhibitor (PARPi) combination regimens have received FDA approval in 2023 for the first-line treatment of metastatic castration-resistant prostate cancer (mCRPC) with deleterious or suspected deleterious BRCA-positive mutations. BRCA mutations make up about 10%–15% of CRPC cases.

Previously, single-agent PARPis were limited only to the second-line setting. These new combination regimens include Merck/AstraZeneca’s Lynparza (olaparib) in combination with abiraterone, Pfizer’s Talzenna (talazoparib) in combination with Pfizer’s Xtandi (enzalutamide), and Janssen’s single-tablet Akeega (niraparib and abiraterone acetate).

Additionally, Pfizer’s Talzenna/Xtandi combination received FDA approval for the first-line treatment of homologous recombination repair (HRR) gene–mutated mCRPC. HRR gene mutations make up about 25% of mCRPC cases.

The attached report examines the differences in approved indication(s), efficacy, costs, and rationale for clinical management of this class, now that all three PARPis have obtained FDA approval for the first-line treatment of mCRPC. The PARPis have other approved indications outside of prostate cancer, including breast, ovarian, and pancreatic cancers; however, this paper focuses on prostate cancer.

It has been recommended that payers implement management strategies for these potentially costly new PARPi combinations that will impact the first-line treatment of prostate cancer.

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