FDA Approves AstraZeneca’s Drug for Generalized Myasthenia Gravis

May 05, 2022 - FDA recently approved AstraZeneca’s Ultomiris to treat adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

The agency based the approval on positive results from the CHAMPION-MG Phase 3 trial, which found that Ultomiris was superior to placebo in the primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 26.

Additionally, the safety profile of Ultomiris was comparable to placebo and consistent with that observed in Phase 3 trials of the drug in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uraemic syndrome (aHUS).

Ultomiris is currently the only approved treatment for long-acting C5 complement inhibitor to treat gMG.

“Since bringing forward the first complement inhibitor, we’ve continued to listen to the community and focused innovation on the needs of gMG patients. We’re proud to deliver on this commitment with today’s approval. Ultomiris, the only long-acting C5 inhibitor, will benefit a broader range of patients, including those with milder symptoms,” Marc Dunoyer, CEO of Alexion, said in the announcement.

“Ultomiris has demonstrated clinical benefit through 60 weeks, with treatment every eight weeks, compared to Soliris every two weeks,” Dunover continued.

gMG is a chronic autoimmune disease that disrupts communication between nerve cells and muscles, causing muscle weakness.

Samantha Masterson, CEO of the Myasthenia Gravis Foundation of America (MGFA), stated that gMG takes a physical and emotional toll on patients living with the disease. But the approval of Ultomiris presents another option as part of their personalized treatment strategies that may offer more convenience and improve muscle weakness.

FDA Approves Bristol Myers’ Drug to Improve Heart Function

FDA recently approved Bristol Myer Squibb’s Camzyos capsules to treat adults with symptomatic New York Heart Association (NYHA) class 2–3 obstructive hypertrophic cardiomyopathy (oHCM).

The agency based the approval on a study that enrolled 220 adults to receive Camzyos or a placebo for 30 weeks. At the end of the study, 37% of participants treated in the Camzyos group improved on the endpoint measuring exercise capacity and symptoms, compared to 17% in the placebo group.

oHCM is a rare disease that occurs when the heart muscle thickens and obstructs blood flow from the heart to the rest of the body. Camzyos reduces heart muscle contraction, which can cause heart failure or block the functioning of the ventricles, and improves exercise capacity and symptoms.  

But FDA noted that patients with a serious intercurrent illness or arrhythmia are at greater risk of developing impaired heart muscle contraction and heart failure with Camzyos and must be monitored with echocardiograms.

Additionally, patients must also avoid certain prescription and over-the-counter medicines that interfere with the drug’s metabolism breakdown.

FDA Approves Mycovia Pharmaceuticals’ Drug for Vulvovaginal Candidiasis

FDA recently approved Mycovia Pharmaceuticals’ Vivjoa (osteseconazole capsules) to reduce the recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC who cannot reproduce.

Vivjoa is the first and only FDA-approved medication for this condition and provides sustained efficacy demonstrated by significant long-term reduction of RVVC recurrent through 50 weeks versus comparators.

The agency based the approval on positive results from the three Phase 3 clinical trials of Vivjoa, two global, pivotal VIOLET studies, and one US-focused ultraviolet study, including 875 patients at 232 sites across 11 countries.

In the two global VIOLET studies, 93.3% and 96.1% of women with RVVC who received Vivjoa did not have recurrent RVVC for the 48-week maintenance period, compared to 57.2% and 60.6% of patients who received placebo.

In the ultraviolet study, 89.7% of women with RVVC who received Vivjoa cleared their initial yeast infection and did not have a recurrence for the 50-week maintenance period compared to 57.1% of those who received fluconazole followed by placebo.

“We celebrate this important milestone for Mycovia, as Vivjoa is the first antifungal in our pipeline to obtain FDA approval and achieves our goal to fulfill a previously unmet medical need among women suffering from RVVC. We are honored to lead this advancement in women’s health,” Patrick Jordan, CEO of Mycovia Pharmaceuticals and partner at NovaQuest Capital Management, said in the announcement.

“We believe the market need for VIVJOA is strong, and we are eager to execute our commercial plans,” Jordan continued.

Mycovia is planning its commercial launch of Vivjoa in the second quarter of 2022.