Insights+: The US FDA New Drug Approvals in April 2022

The US FDA has approved 6 NDAs in Apr 2022, leading to treatments for patients and advances in the healthcare industry. The CDER and CBER approved 26 novel products in 2022
In Apr 2022, the major highlights drugs were Vivjoa for Recurrent Vulvovaginal Candidiasis, Cuvrior for Stable Wilson’s Disease, Igalmi for Acute Treatment of Agitation Associated with Schizophrenia or Bipolar I or II Disorder
PharmaShots has compiled a list of a total of 6 new drugs approved by the US FDA in April 2022

Vijoice

Active ingredient: alpelisib Approved: April 7, 2022

Company: Novartis Disease: PIK3CA-Related Overgrowth Spectrum

The US FDA approval was based on RWE from the (EPIK-P1) study evaluating Vijoice in adult & pediatric patients aged ≥2yrs. with PIK3CA-related overgrowth spectrum
The results showed a 74% reduction in target lesion volume with a mean reduction of 13.7%, 27% with a confirmed response with ≥20% reduction in PROS target lesion volume @24wk., no patients experienced disease progression at the time of primary analysis
Additionally, improvements in pain (90%), fatigue (76%), vascular malformation (79%), limb asymmetry (69%) & disseminated intravascular coagulation (55%). The company provides a patient support program including assistance to access medication, financial resources & continued education

Igalmi

Active ingredient: dexmedetomidine Approved: April 7, 2022

Company: BioXcel Therapeutics Disease: Agitation

The approval was based on the (SERENITY I & II) studies evaluating Igalmi vs PBO in patients with agitation associated with schizophrenia & bipolar I or II disorder. The product is expected to be available in the US in Q2’22
Both trials met its 1EPs & 2EPs i.e., both (120 & 180mcg) doses showed improvements from baseline along with a high response rate & demonstrated a rapid onset of action @20min. for both 180 & 120mcg doses in (SERENITY II) study while 20 & 30min. in (SERENITY I) study, respectively
The results from the P-III (SERENITY II) trial in bipolar disorders were published in the JAMA. Igalmi can be self-administrated by patients under the supervision of a healthcare provider

Epsolay

Active ingredient: benzoyl peroxide Approved: April 25, 2022

Company: Sol-Gel Technologies and Galderma Disease: Inflammatory Lesions of Rosacea

The approval was based on the two P-III trials to evaluate Epsolay vs vehicle cream in patients with inflammatory lesions of rosacea
The coprimary EPs in both trials were the proportion of patients with treatment success & the absolute change from baseline in lesion counts @12wk. the therapy was effective @ 4wks. of treatment, 70% vs 38-46% reduction in inflammatory lesions of rosacea & 50% were clear or almost clear over 38-46% with PBO @12wk. Post-hoc analysis of lesion count & IGA success demonstrated a greater treatment effect as early as 2wk.
In the OLE study, 73% were clear or almost clear @52wks. Epsolay (benzoyl peroxide, cream 5%) uses microencapsulation technology & is patent protected until 2040

Vivjoa

Active ingredient: oteseconazole Approved: April 28, 2022

Company: Mycovia Disease: Recurrent Vulvovaginal Candidiasis

The approval was based on 3 P-III trials i.e., two (VIOLET) & 1 US (ultraviolet) study evaluating oteseconazole vs PBO in 875 patients with RVVC at 232 sites across 11 countries. Vivjoa is expected to be available in Q2’22
The results from 2 (VIOLET) studies showed that (93.3% & 96.1% vs 57.2% & 60.6%) of women achieved a reduction of RVVC recurrence for 48wk. maintenance period. In the (ultraVIOLET) study, 89.7% vs 57.1% of women cleared initial yeast infection & did not have a recurrence for 50wk. maintenance period along with sustained efficacy
Vivjoa is the 1st US FDA approved product & an azole antifungal that was indicated to reduce the incidence of RVVC in females

Camzyos

Active ingredient: mavacamten Approved: April 29, 2022

Company: BMS Disease: NYHA class II-III obstructive HCM

The approval was based on the P-III (EXPLORER-HCM) trial to evaluate Camzyos vs PBO in a ratio (1:1) in 251 adult patients with NYHA class II-III obstructive HCM to improve functional capacity & symptoms
The results showed that 37% vs 17% of patients achieved the composite 1EPs @30wk., greater improvement across all 2EPs including mean change from baseline post-exercise LVOT peak gradient & pVO2, improvement of NYHA class ≥1 (65% vs 31%)
Mean change from baseline in KCCQ-23 † CSS, KCCQ-23 TSS & KCCQ-23 PL, mean resting LVEF was 74% at baseline in both groups, 6% vs 2% experienced reversible reductions in LVEF to <50%. The company offers multiple programs and resources along with patients support

Cuvrior

Active ingredient: mavacamten Approved: April 30, 2022

Company: Orphalan Disease: Stable Wilson’s Disease

The US FDA has approved Cuvrior for the treatment of adult patients with stable WD who are de-coppered & tolerant to penicillamine
The approval was based on data on the P-III (CHELATE) study to evaluate Cuvrior vs penicillamine in 53 adults with WD. The trial met its primary efficacy EPs i.e., Cuvrior was found to be non-inferior to penicillamine & showed that patients treated with Cuvrior had a similar mean NCC level to penicillamine @36wks., Cuvrior treated patients had a lower mean 24hr. urine copper excretion than penicillamine
Cuvrior is a functionally scored tablet that contains 300mg trientine tetrahydrochloride. The product is expected to be available in early 2023