When Neurocrine Bioscience won a nod from the FDA for Ingrezza (valbenazine) a couple of weeks ago as the first approved treatment for adults with tardive dyskinesia (TD), the company didn’t divulge a price but said it would be competitive with Teva’s Austedo (deutetrabenazine), which had just been approved for chorea associated with Huntington’s disease and could be approved later this year for TD as well. Austedo has a list price of $60,000 per year. Neurocrine has since given Ingrezza a price tag of $5,275 for a 30-count bottle of 40 mg capsules, yielding an annual cost of about $64,000—higher than most industry analysts were anticipating—and that’s if patients only have to take 40 mg daily to treat their symptoms. Should they require a daily dose of 80 mg, the annual cost would exceed $127,000—at least until Neurocrine gets FDA approval for its 80 mg capsules, which is expected to occur by year-end. The company has said the 80 mg formulation would be “priced substantially similar” to the 40 mg capsules. According to FiercePharma, Neurocrine said it expects Teva to charge closer to $70,000 per year for Austedo as a treatment for TD if it is approved for that indication, based on the drug’s per-milligram cost and the dosage used in clinical trials.
Valeant Pharmaceuticals has priced Siliq (brodalumab), the injectable interleukin-17 (IL-17) inhibitor approved in February for adults with moderate to severe plaque psoriasis, at $3,500 per month, or $42,000 annually. Valeant says the cost of Siliq is the lowest on the market for this new generation of drugs, which compete with well-established TNF-alpha blockers such as AbbVie’s Humira (adalimumab). As the third IL-17 inhibitor to be approved for this indication, Siliq will have to play catch-up with Novartis AG’s Cosentyx (secukinumab) and Eli Lilly & Co.’s Taltz (ixekizumab), approved in 2015 and 2016, respectively. Additionally, Siliq was approved with a black box warning for an increased risk of suicidal thoughts and behavior, as well as restricted access through a risk evaluation and mitigation strategy program—which neither of the other two approved drugs required. Amgen originally partnered with AstraZeneca to develop brodalumab but gave the rights to the drug back to AstraZeneca when the suicide risk became evident in clinical trials. AstraZeneca subsequently out-licensed the drug to Valeant for an upfront payment of $100 million plus milestones. Valeant will also have to share U.S. profits for Siliq with AstraZeneca.
The FDA expanded the approved indications for Bayer’s Stivarga (regorafenib) to include the treatment of patients with hepatocellular carcinoma who have stopped responding to initial treatment with Bayer’s Nexavar (sorafenib). The move makes Stivarga the first drug to be approved for liver cancer in nearly a decade. Stivarga was already approved as a second-line treatment for colorectal cancer and gastrointestinal stromal tumors.
Patients with newly diagnosed acute myeloid leukemia (AML) who have a certain genetic mutation now have a new targeted treatment option. The FDA approved Novartis’ Rydapt (midostaurin) in combination with chemotherapy for patients with the FLT3 genetic mutation, as detected by an FDA-approved companion diagnostic. Medscape reported that an estimated 30 percent to 35 percent of patients with AML have this mutation, which is associated with a worse prognosis. Rydapt is the first new drug to be approved for AML since 1990. The FDA also approved Rydapt for use in treating adults with the rare blood disorders aggressive systemic mastocytosis and systemic mastocytosis with associated hematologic neoplasm or mast cell leukemia.
Certain women with osteoporosis have a new treatment option. The FDA approved Radius Health’s Tymlos (abaloparatide) for postmenopausal women with osteoporosis who are at high risk for fracture, either because they have of a history of osteoporotic fracture or they have multiple risk factors for fracture. The drug, administered subcutaneously once a day, is also approved for patients who have not responded to or cannot tolerate other available therapies for osteoporosis. Radius Health said Tymlos is the first new anabolic treatment to be approved in the U.S. for postmenopausal women with osteoporosis in almost 15 years.
BioMarin Pharmaceutical received FDA approval of its Brineura (cerliponase alfa) as a treatment for children who have a certain form of Batten disease known as CLN2 disease, which the company describes as “an ultra-rare, rapidly progressive fatal brain condition.” CLN2, also known as tripeptidyl peptidase 1 (TPP1) deficiency, is a lysosomal storage disorder. Children who have CLN2 disease usually start to experience seizures between the ages of 2 and 4 years. Most lose the ability to walk and talk by the time they are 6 years old, and death occurs in many cases between the ages of 8 and 12. Brineura, an enzyme replacement therapy, is the first drug approved to treat CLN2 disease; it is indicated to slow the loss of ambulation in symptomatic children aged 3 years or older. The drug is infused directly into the fluid surrounding the brain via intraventricular administration. BioMarin will be required to further evaluate the safety of Brineura in children younger than 2 years and will have to conduct a long-term safety study of the drug. BioMarin anticipates launching Brineura by early June and has priced it at $702,000 per year, making it one of the costliest orphan drugs on the market. The company said there are several reasons for the extremely high price, including the exceptionally small patient pool available for developing and testing the drug, and the need for “specialized” manufacturing.
Takeda Pharmaceutical Co. received accelerated FDA approval for Alunbrig (brigatinib) as a treatment for patients with anaplastic lymphoma kinase-positive (ALK+) metastatic non-small cell lung cancer whose disease has progressed during treatment with Pfizer and EMD Serono’s Xalkori (crizotinib), or those who cannot tolerate Xalkori. Takeda gained the orphan drug when it acquired Ariad Pharmaceuticals earlier this year. At the time, Takeda said Alunbrig had the potential to achieve peak annual sales of more than $1 billion. Takeda has not said what it will charge for the drug but plans to launch it “as soon as possible,” FiercePharma reported. It will have to compete with Genentech’s Alecensa (alectinib) and Novartis’ Zykadia (ceritinib).