April 2022 Drug Launch Highlights

Camzyos

28-Apr-2022

Bristol Myers Squibb announced the approval of the mavacamten, marketed as Camzyos, for the treatment of New York Heart Association Class II-III obstructive hypertrophic cardiomyopathy (HCM). This makes mavacamten the first approved pharmaceutical which targets the causative mechanisms for obstructive HCM, by inhibiting cardiac myosin. Mavacamten works as a selective, reversible inhibitor and its efficacy was demonstrated in the EXPLORER-HCM Phase III trial. Over 37% of patients taking mavacamten showed significant improvement of mixed venous oxygen tension, or improvement in the New York Heart Association Class of obstructive HCM. This compares very favorably compared to the 17% of the placebo control group that showed similar improvements, demonstrating the potential for the drug to serve an unmet need. Patients also showed improvements across a spectrum of other endpoints, including improvements in post-exercise left ventricular outflow tract gradients and better clinical summary scores overall. Adverse events noted for the pharmaceutical include systolic dysfunction in patients with reduced left ventricular ejection fractions – for which the drug has received a boxed warning and mandates monitoring for heart function prior to, and during, administration. Other side effects can include dizziness and syncope. Due to these, the pharmaceutical has been made available only through a Risk Evaluation and Mitigation Strategy program, particularly due to its potential for negative drug interactions with other active ingredients which can affect the heart. However, with obstructive hypertrophic cardiomyopathy being the most common genetic heart disease in the USA, the drug is expected to bring much needed relief to a significant patient population – as stated by Samit Hirawat, Chief Medical Officer for BMS:

““This approval builds on decades of cardiovascular leadership and reflects our steadfast commitment to people impacted by cardiovascular disease. We are proud to bring this first-of-its kind medicine to patients, which may help to address an unmet need in the U.S. in the symptomatic NYHA class II-III obstructive HCM treatment landscape.”

Cuvrior

28-Apr-2022

Orphalan announced the approval of trientine tetrahydrochloride, marketed as Cuvrior, for the treatment of individuals with stable Wilson’s disease. Wilson’s disease is a rare genetic disorder which affects copper transport throughout the body, typically resulting in copper accumulation in the liver, brain, eyes and other tissues. Due to the progressive nature of the disease, treatment is necessary to prevent disability, hepatic damage, dysfunction of the central nervous system and, ultimately, death. The primary mode of treatment is the reduction of copper levels in the body. The current standard-of-care in America is treatment with penicillamine, which binds to copper and directs its excretion through urine – although one-third of patients will eventually develop intolerance to penicillamine, highlighting the need for alternative options. Cuvrior has demonstrated non-inferiority to penicillamine in its phase III CHELATE trial, and is already approved for use in Europe under the marketing designation Cuprior. It should be noted that Cuvrior did not show comparable efficacy in patients who were already intolerant to penicillamine. The drug is expected to launch in early 2023, and will hopefully provide a treatment option that is less likely to lead to long-term intolerance – as reiterated by Michael Skilsky, MD, Director of the Center for Excellence for Wilson Disease at Yale University:

“As a physician, I have seen first-hand how Wilson’s disease impacts the lives of patients and, until now, there have been few effective long-term treatment options available. The approval of Orphalan’s Cuvrior™ by the FDA is backed by positive data from Orphalan’s multicenter, multinational CHELATE trial – the first head-to-head controlled study of a new trientine salt versus penicillamine. For patients in need, Cuvrior™ represents a well-tolerated and effective alternative to penicillamine, the current standard of care.”

VIVJOA

26-Apr-2022

Mycovia Pharmaceuticals announced the approval of its novel product, oteseconazole, marketed as VIVJOA – a first-in-class treatment for recurrent vulvovaginal candidiasis, also known as a vaginal chronic yeast infection. Oteseconazole is an azole antifungal, and is primarily aimed at treating the disease in females with a history of recurrent candidiasis who are not of reproductive potential. Considering that nearly three quarters of women will suffer from vulvovaginal candidiasis, up to 9% of which will then experience recurrent symptoms, the clinical need for a more diverse portfolio of treatment options cannot be understated. Oteseconazole is an inhibitor for fungal CYP51, exerting its antifungal effects by compromising cell wall integrity, while demonstrating lower binding affinities for human CYP enzymes. VIVJOA is Mycovia’s first approved product, and the only treatment to target recurrent vulvovaginal candidiasis – which can present symptoms such as chronic itchiness, discomfort, inflammation and irritation. In its two global VIOLET studies, VIVJOA demonstrated no symptom recurrence in over 93% of patients – a significantly superior observation compared to the placebo groups, for which no more than 60% experienced non-recurrence in the 48-week study window. Data on human females with reproductive potential is unavailable, but studies in rats have shown that the drug can cause negative interactions. Stephen Brand, Chief Development Officer at Mycovia, highlighted the future possibilities in treating the disease in a forward-looking statement:

“A medicine with VIVJOA’s sustained efficacy combined with the clinical safety profile has been long needed, as until now, physicians and their patients have had no FDA-approved medications for RVVC. We are excited to be the first to offer a medication designed specifically for RVVC, a challenging and chronic condition that is expected to increase in prevalence over the next decade.”

Vijoice

6-Apr-2022

Novartis announced the accelerated FDA approval of Vijoice, a first-in-class treatment for PIK3CA-related overgrowth spectrum (PROS) in adult as well as in pediatric populations. This makes Vijoice the only treatment available for patients with PROS, a spectrum of rare conditions which causes blood vessel anomalies and overgrowths, affecting 14 people per million. The approval comes after positive results from the EPIK-P1 study which demonstrated significant reductions in target lesion volume and improvement across other PROS symptoms, while no patients on the treatment arm of the study exhibited any disease progression during the 24 week study window. Adverse events included diarrhea, stomatitis and hyperglycemia – although none of these were observed in more than 16% of the population. The approval marks further developments in the space of rare disease, offering treatment options for populations which had none, as stated by Kristen Davis, Executive Director of the CLOVES Syndrome Community:

“Today’s approval of the first treatment for PROS offers hope for a better quality of life to patients and families affected by these rare conditions. PROS conditions can be debilitating and disabling and can result in disruptions to everyday activities. Until today, often the only treatment options for patients were surgical or interventional radiology procedures.”

Nick Zoukas, Former Editor, PharmaFEATURES