Ravulizumab-CWVZ

Alexion’s newest film premiered at the Myasthenia Gravis Foundation of America’s international conference in May, where it was “extremely well received” by both patients and caregivers, Christophe Hotermans, M.D., Ph.D., Alexion’s head of global medical affairs, reported. A year after debuting the “Rare Connections” series with a short film about patients’ experiences with neuromyelitis optica spectrum disorder (NMOSD), AstraZeneca’s rare disease unit is turning the cameras on generalized myasthenia gravis (gMG).Alexion’s “Rare Connections in gMG” film stars three people with the neurological autoimmune disease, for which AstraZeneca markets Ultomiris and Soliris.Throughout the nearly eight-minute video, the film’s stars are shown writing—and reading aloud—journal entries about their gMG and the support they’ve received from loved ones, before those friends and family members are invited to read the entries themselves.The film ends with shots of the gMG patients walking in during their loved ones’ filming sessions to surprise them with hugs and share their gratitude in person.As with the NMOSD-focused film, “Rare Connections in gMG” is aimed at countering the “relatively low” awareness of the rare disease, according to Christophe Hotermans, M.D., Ph.D., Alexion’s head of global medical affairs.“The reason for this movie is really to highlight the fluctuating and often invisible symptoms of the disease,” Hotermans said in an interview with Fierce Pharma Marketing. “And the goal is really to foster connection with the gMG community and raise awareness about the disease’s physical and emotional impact.” With that in mind, the target audience for the film is, in a word, “everybody,” he said.Not only can the film offer solace to gMG patients who feel isolated and misunderstood, he suggested, but it may also help educate their caregivers, loved ones, neighbors and the general public about the reality of life with gMG. That broad swath even includes healthcare providers, as Hotermans said that Alexion has heard from patients who “experience symptoms that are not in textbooks” and so aren’t necessarily being addressed by their doctors.As for the content of the film, Hotermans said Alexion’s approach was “to share problems that we’ve heard from the patient community and that they really wanted to highlight and that would be representative of what they experience.”To that end, the film’s stars were largely able to shape their onscreen narratives, he said. Deanna, for example, wanted to shed light on her feelings of being misunderstood by her doctor, especially as her symptoms worsen as the day goes on and so may not seem as severe during morning appointments. Another patient, Nick, took years to understand that his lifelong health issues were due to gMG and so chose to focus on the importance of reaching a diagnosis. Finally, Marta opted to highlight “the unpredictability of the disease,” per the exec.The film premiered at the Myasthenia Gravis Foundation of America’s international conference in May, where it was “extremely well received” by both patients and caregivers, Hotermans reported.That represents a key performance indicator for the production, as Hotermans noted that its success will first and foremost be measured by how well it resonates with the gMG community. And, in the longer term, he said, “ultimately, success is really reflected in increased understanding and the meaningful dialogue we spark.” 

AstraZeneca is planning to present gefurulimab data at a medical meeting and share the results with regulators. \n A phase 3 study of AstraZeneca’s gefurulimab has hit its primary and all secondary endpoints, teeing up talks with regulators about the potential blockbuster generalized myasthenia gravis (gMG) treatment.AstraZeneca listed gefurulimab among candidates it expects to generate sales of between $1 billion and $3 billion at its investor day last year. The forecast rests on the belief that the weekly, self-administered subcutaneous nanobody C5 inhibitor can unlock an earlier, broader population than AstraZeneca’s existing gMG drug Ultomiris, which is given as a one-hour infusion and is feeling the pressure in a competitive market. The phase 3 trial randomized 260 people with anti-acetylcholine receptor antibody-positive gMG to take gefurulimab or placebo. After 26 weeks, people on gefurulimab performed significantly better on a gMG scale that assesses the ability to perform daily activities, achieving the primary endpoint of the study.Marc Dunoyer, chief of AstraZeneca’s rare disease unit, said in a statement that the data “reinforce the established safety profile and efficacy of C5 inhibition and show the potential for gefurulimab as a first-line biologic, with the convenience of a self-administered option.” The trial’s secondary endpoints assessed how people performed on other gMG scales and evaluated the proportion of patients who were classed as responders. AstraZeneca is yet to share any data, saying only that the trial met all its endpoints, gefurulimab was well tolerated and no new safety signals were seen.AstraZeneca is planning to present the data at a medical meeting and to share the results with regulators. The company has identified gefurulimab as a product that can drive growth through 2030 and beyond by opening up more of the market. AstraZeneca sells Ultomiris in gMG, but it is used by more advanced patients. Competitive pressure in gMG offset growth of Ultomiris in other indications in the first quarter.“Ultomiris has a key position in this market, but, obviously, other mechanisms have an attraction for people who are switching from the steroids or immunosuppressant as a first-line treatment. Ultomiris is also competing in that segment, but it’s not the only mechanism available,” Dunoyer said in April.The gMG market has grown in recent years as FcRn inhibitors have won approval. Led by argenx’s Vyvgart Hytrulo, the drug class has given gMG patients self-administered subcutaneous treatment options.