Highlights in Drug Approvals May-June 2022

Amvuttra

13 June 2022

The leading company in the RNA interference (RNAi) space, Alnylam, has announced the FDA approval of Amvuttra. An RNA therapeutic based on vutrisiran, Amvuttra is administered once every three months by subcutaneous injection for the treatment of hereditary transthyterin-mediated (hATTR) amyloidosis in adults. hATTR is a hereditary disease which can prove rapidly progressive and fatal, with multiple neuropathy complications and scant treatment options. The approval comes after the HELIOS-A Phase 3 trial, with more than 50% of patients experiencing halting or reversal of clinical symptom – with treatment showing a 2.2 mean decrease in the modified Neuropathy Impairment Score + 7 at 9 months. Additionally, Amvuttra met all secondary endpoints, including improved quality of life and walking scores. The safety profile of the drug seems promising, with no drug-related discontinuations – indicating high tolerability. The approval continues to highlight the potential for RNA-based therapeutics, and their promise to open up vast swaths of undruggable targets and address clinical burdens. Similar sentiments were expressed by Isabelle Lousada, Founder and CEO of the Amyloidosis Research Consortium:

“Today we celebrate the FDA’s approval of vutrisiran, a welcomed treatment option for hATTR amyloidosis patients experiencing the challenges of the polyneuropathy of the disease […] With this approval, Alnylam has expanded treatment options that may support improvements in quality of life, providing hope for patients and families in the amyloidosis community.”

Vtama

23 May 2022

Dermavent Sciences announced the approval of VTAMA earlier this year, marking the first time in 25 years where a novel topical chemical agent is approved for use in psoriasis. Conjointly, VTAMA is the only steroid-free medication in its class, working as an aryl hydrocarbon receptor agonist. Over 40% of Phase 3 study participants demonstrated complete disease remission, with months-long remission subsequent to the trial. Self-reporting questionnaires indicated high patient satisfaction with the therapy. With 57.8 per 100,000 people worldwide suffering from psoriasis, innovating novel and effective therapies to treat the disease is an urgent need. Philip M. Brown, M.D., J.D., Chief Medical Officer at Dermavent, also highlighted the potential for using VTAMA in other indications and therapeutic areas:

“We believe VTAMA cream has the potential to make a meaningful difference in the lives of patients with plaque psoriasis as well as their healthcare providers […] We are continuing to leverage our experience and insights with the active ingredient, tapinarof, and potentially other AhR molecules, for potential application to other conditions in dermatology and additional inflammatory and immunology indications.”

Mounjaro

13 May 2022

Eli Lilly announced an FDA approval for Mounjaro (tirzepatide), an innovative treatment for Type 2 Diabetes. Mounjaro is currently the only therapy based on a glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide 1 (GP1) receptor agonist. In conjunction with diet and exercise, the treatment is aimed at increasing glycemic control by activating GIP and GP1 receptors, resulting in incretin hormones which stimulate insulin secretion. The Phase 3 trial for the treatment, SURPASS, reported superior A1C reductions as a primary endpoint; weight loss acted as a secondary endpoint, which was met. Though Mounjaro is not indicated for weight loss, treated participants in the trial lost between 5-15kg on average. The company announced further results in June, after the approval of the drug, reinforcing the efficacy of the therapeutic. The safety profile of the drug is well-tolerated, although mouse models demonstrated the possibility of thyroid C-cell tumors – resulting in a boxed warning for this risk and contraindications for patients with family histories of related conditions. Pancreatitis has also been reported, and is another contraindication for the drug. Lilly remains committed to further exploring the space for incretin-based therapies and the intersection between glucose control and weight loss, as reiterated by Laura Fernández Landó, MD, associate vice president, Medical, Lilly Diabetes:

“Lilly is proud to present new mechanism of action data and new analyses of the results that Mounjaro delivered throughout the SURPASS program at the ADA’s Scientific Sessions, helping us further evaluate how Mounjaro can help adults living with type 2 diabetes manage key aspects of their disease […] Exploring factors such as how quickly Mounjaro can help lower A1C and weight, or the relationship between those two measures throughout the SURPASS program, is important as we begin to bring Mounjaro to people living with type 2 diabetes.”

Voquenza

03 May 2022

Phantom Pharmaceuticals announced the approval of Voquenza by the FDA early in May, marking the first time a product with vonoprazan as an active ingredient is marketed in the United States. Voquenza is sold as either a triple or dual mixture of vonoprazan with amoxicillin and clarithromycin, or vonoprazan with amoxicillin. The therapy is indicated for the treatment of infection with Helicobacter pylori, a bacterium known to cause peptic ulcers, and even gastric cancers. Vonoprazan works as a potassium-competitive acid blocker, suppressing acid levels in the stomach and allowing antibiotics to work more effectively. With H. pylori eradication levels dropping below 80% in America, and nearly 115 million affected people, novel tools for lowering the burden of the infection have the capacity to drastically ease the burden of downstream complications and clinical needs. The therapy was approved based on results from a Phase 3 trial called PHALCON-HP, the largest registrational trial ever conducted on H. pylori in the United States. Voquenza demonstrated improved eradication rates over standard-of-care routines, even in clarithromycin-resistant H. pylori strains, while maintaining comparable safety profiles. Terrie Curran, Chief Executive Officer at Voquenza, highlighted the importance of continuing the fight against H. pylori:

“H. pylori eradication rates continue to decline in part due to antibiotic resistance, inadequate acid suppression, and complex treatment regimens resulting in treatment failures and complications for patients. New therapies that have the potential to address the limitations of current treatments are needed and we look forward to bringing these innovative vonoprazan-based treatment options to the millions of H. pylori sufferers in the U.S.”