At this month’s meeting, the EMA’s Committee for Medicinal Products for Human Use issued recommendations of approval for 10 drugs. They include:
- Oxlumo (lumasiran), Alnylam’s RNAi candidate that snagged priority review from the FDA back in May. In June, the biotech released pivotal data which showed that 21 of 25 patients with PH1 (primary hyperoxaluria type 1), a rare liver condition, saw their oxalate levels fall to near the normal range after being treated with lumasiran, and just over half had numbers inside the normal range. That was compared to 0% in the placebo group, according to Alnylam.
- Kite’s Tecartus (KTE-X19), a CAR-T therapy approved by the FDA in July to treat adult patients with relapsed or refractory mantle cell lymphoma, a rare form of NHL. Ken Takeshita, head of global development at Gilead subsidiary Kite Therapeutics, boasted that the drug’s data were better than the company’s CAR-T drug Yescarta, which was approved in 2017. A company spokesperson said in July that the drug will be sold for $373,000.
- Novartis’ Leqvio (inclisiran), the LDL-C drug that was formerly a Medicines Company candidate. An August read-out from two Phase III trials showed consistency in efficacy and safety among patients with both hyperlipidemia and atherosclerotic cardiovascular disease despite statin therapy, according to the company. Around 88% of patients hit 50% reduction of LDL-C in at least one point during the 17-month study. After the 17-month mark, almost two-thirds of patients (66.4%) had a 50% reduction compared to 2.5% percent in the placebo group.
- Orchard Therapeutics’ Libmeldy for metachromatic leukodystrophy
- Janssen’s Rekambys for human immunodeficiency virus type 1 (HIV-1)
- ViiV Healthcare’s Vocabria for HIV-1
- Zogenix’s Fintepla for the treatment of seizures associated with Dravet syndrome
- Aimmune’s Palforzia for desensitising children and adolescents to peanut allergy
- AstraZeneca’s Trixeo Aerosphere for the maintenance treatment of chronic obstructive pulmonary disease
- Mylan’s lenalidomide for the treatment of multiple myeloma and follicular lymphoma
— Nicole DeFeudis
Sanofi’s big cancer play gets the OK from UK price watchdog
Sanofi won FDA and EMA approval for their first homegrown cancer drug in a decade earlier this year. Today, they secured another victory as a UK drug pricing watchdog cleared the therapy for use in the UK.
The multiple myeloma drug nabbed the recommendation after showing a progression-free survival of 11.5 months in pivotal trials, compared to 6.5 months for the standard-of-care. Because full data on survival benefit are not yet available, the council, known as NICE, did not recommend it for “routine” NHS coverage. However, it recommended the drug for inclusion in the Cancer Drugs Fund, a fund set up to help cover costs of cancer drugs that are not cost effective.
Sanofi is taking on J&J and their multiple myeloma blockbuster Darzalex as they market their first big oncology win in years.
– Jason Mast