Biospace shared the summary of Clarivate Analytics’ report “Cortellis Drugs to Watch,” in which seven new drugs identified as blockbusters by 2023, here is the list;
#1. AbbVie’s Upadacitinib. On December 20, 2018, AbbVie submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA)and a marketing authorization application (MAA) to the European Medicines Agency (EMA)for upadacitinib. The FDA accepted the NDA on February 19 under Priority Review, with a regulatory decision expected in the third quarter. The drug is an oral investigational JAK1-selective inhibitor to treat adults with moderate to severe rheumatoid arthritis.
The NDA and MAA are built on data from the company’s SELECT Phase III rheumatoid arthritis program that studied more than 4,000 rheumatoid arthritis patients in five Phase III trials. The drug met all primary and ranked secondary endpoints.
#2. Novartis/AveXis’ Zolgensma. Novartis and its subsidiary, AveXis, announced the FDA had accepted its Biologics License Application (BLA) for Zolgensma (onasemnogene abeparvovec-xxxx) on December 3, 2018, for spinal muscular atrophy (SMA) Type 1. A target action date is set for May 2019. SMA is a deadly neuromuscular disease caused by a defective or missing SMN1 gene. It typically affects infants, who quickly lose the motor neurons that control muscle functions like breathing, swallowing, speaking and walking. Without treatment, death usually occurs before two years of age.
The drug is also being reviewed in Japan and Europe. The regulatory target date in Japan is in the first half of this year. The EMA decision is expected in mid-2019. Japan and the EU have given the drug Sakigake and Prime designations, equivalent to the FDA’s Breakthrough Therapy designation, which the FDA has also granted.
#3. AstraZeneca’s Roxadustat. AstraZeneca and its collaboration partner FibroGen received marketing approval in China for their roxadustat for anemia caused by chronic kidney disease (CKD) in December 2018. The drug is an oral, small molecule, hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) indicated for patients on hemodialysis or peritoneal dialysis. The drug increases the levels of erythropoiesis by increasing erythropoietin production and improving iron regulation. The drug is expected to launch in China in the second half of this year. FibroGen and Astellas Pharma are working to develop and commercialize the drug in Japan, Europe, the Commonwealth of Independent States, the Middle East, and South Africa. AstraZeneca and FibroGen are developing it in the U.S., China, and other global markets.
#4. Alexion’s Ultomiris. The FDA approved Alexion Pharmaceuticals’ Ultomiris (ravulizumab-cwvz) in December 2018 for adults with paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare blood disorder characterized by complement-mediated destruction of red blood cells. This was significantly ahead of the FDA’s target action date of February 18, 2019. The drug is also being reviewed in Europe and Japan.
“We are proud to bring Ultomiris to patients suffering from this devastating disease less than a year after reporting our positive Phase III data,” stated John Orloff, executive vice president and head of Research & Development at Alexion in December. “Immediate and complete C5 inhibition with Ultomiris, sustained for eight weeks, can provide meaningful benefits for patients and their families.”
#5. AbbVie and Boehringer Ingelheim’s Skyrizi. Skyrizi (risankizumab) is part of a collaboration between Boehringer Ingelheim and AbbVie. The drug is to treat moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. On March 1, the EMA’s Committee for Medicinal Products for Human Use (CHMP) gave the drug a positive recommendation. The data for the submission came from the companies’ Phase III psoriasis program that studied more than 2,000 patients with moderate to severe plaque psoriasis in four Phase III trials. The drug is also being evaluated by the FDA.
#6. Aimmune Therapeutics’ AR-101. On March 18, the FDA accepted Aimmune Therapeutics’ Biologics License Application (BLA) for AR101 as a treatment to decrease the risks of anaphylaxis after accidental exposure to peanuts. The drug received Breakthrough Therapy Designation in June 2015, as well as Fast Track Designation. In November 2018, the company published results from its Phase III PALISADE clinical trial in the New England Journal of Medicine (NEJM). AR101 is 12 percent defatted peanut flower using a Good Manufacturing Process (GMP). The idea is that it can be used to expose people with peanut allergies in controlled amounts so they can develop a tolerance for the allergen. In the trial, about two-thirds tolerated a single dose of at least two peanuts, and about 85 percent tolerated the equivalent of three to four peanuts. About half tolerated the highest levels, a single dose equal to three to four peanuts and a total of seven to eight total exposure. Although there was a favorable safety profile, there were adverse side effects in more than 95 percent of patients, almost all mild or moderate.
#7. Bluebird bio’s LentiGlobin. On December 3, 2018, bluebird bio announced new datafrom patients in Group C of its ongoing Phase I/II trial of LentiGlobin gene therapy for sickle cell disease (SCD). LentiGlobin is a one-time gene therapy that is in ongoing clinical trials all over the world. Both the FDA and EMA have granted it orphan drug status and Fast Track designation. The company plans to launch a Phase III study of the therapy in SCD this year. In the early trials, the therapy appeared effective in treating SCD.
“After patients with sickle cell disease were treated with LentiGlobin they began to produce gene-therapy derived HbAT87Q, which was associated with lower levels of sickling hemoglobin, the type of hemoglobin that damages red blood cells,” stated David Davidson, bluebird bio’s chief medical officer. “These clinical findings were consistent with results in newly developed exploratory assays used to evaluate patient samples that demonstrated reduction of HbS in most red blood cells, and a reduction in sickling comparable to sickle-trait, suggesting the potential for LentiGlobin to fundamentally improve the underlying red blood cell pathology responsible for the clinical consequences of sickle cell disease.”