Novartis’ and Gilead’s CAR-Ts, Alnylam’s Onpattro and others rack up EU approvals

Not that it’s a competition, but after the U.S. FDA pulled off several nods last week, its European counterpart has matched up with its own party of drug approvals. As Gilead and Novartis won key green lights for their CAR-Ts on Monday, Jazz Pharma, Ultragenyx, AstraZeneca and Amgen also earned their own go-ahead stamps this week.

Novartis’ Kymriah and Gilead’s Yescarta—the world’s first two CAR-T therapies—kicked off the week at the European Commission. Kymriah is now allowed to be marketed in Europe for B-cell acute lymphoblastic leukemia (ALL) and treatment-resistant diffuse large B-cell lymphoma (DLBCL), while rival Yescarta was approved to treat DLBCL and primary mediastinal large B-cell lymphoma.

The Gilead drug is currently leading the space with $68 million in second-quarter sales, as compared to Kymriah’s $16 million. One issue pulling Novartis back is manufacturing constraint. As a result, Novartis said it can initially only sell the med to children with ALL, but it’s investing CHF90 million ($91.5 million) over three years to ramp up production capacity in Switzerland.

Gilead is taking a similar step to make the complex drug geographically closer to the European patients. It hopes a new manufacturing facility in the Netherlands dedicated to cell therapy will be fully operational in 2020.

RELATED: With Kymriah nod in EU, Novartis is building a plant with up to 450 jobs

In a quick EU follow-up to an FDA nod, Alnylam was granted marketing authorization for Onpattro. The FDA approved it several weeks ago. The first approved RNAi therapy treats transthyretin amyloidosis (ATTR) polyneuropathy. But it could soon face a rival, as Pfizer just reported positive data for its ATTR cardiomyopathy candidate tafamidis. As analysts see it, both drugs could tap into the other’s territory.

Another rare disease nod went to Ultragenyx’s Mepsevii, designed for an ultra-rare genetic condition called mucopolysaccharidosis VII. The drug became the company’s first product last November when it was approved by the FDA. Evercore ISI analyst Steven Breazzano at that time predicted peak sales of about $75 million. 

In a second win for Novartis this week, a Tafinlar-Mekinist combo earned its third EU indication as post-surgery treatment of stage III patients with BRAF V600 mutant melanoma, following a nod last year in non-small cell lung cancer. The former GlaxoSmithKline drug, which Novartis gained through their 2015 asset swap, is on track to reach blockbuster status this year, having put up double-digit growth, or $551 million revenue, in the first six months of 2018.

RELATED: Pfizer's tafamidis score still leaves 'door open' for Alnylam rival: analysts

Meanwhile, two drugs secured indication expansions for pediatric use. Amgen’s leukemia drug Blincyto just turned its conditional EU nod in relapsed or refractory B-cell precursor ALL into a full one two months ago. Now, the bispecific T-cell engager and GlaxoSmithKline’s asthma drug Nucala are both allowed in children. In addition, Eisai extended use of epilepsy drug Inovelon (marketed as Banzel in the U.S.) to children as young as one year old.

Jazz Pharma nabbed EU approval for Vyxeos in certain types of high-risk acute myeloid leukemia (AML). The chemo combo therapy was approved by the FDA a year ago, based on clinical evidence that it separately beat the two chemo drugs included in it.

For the second quarter, Vyxeos registered sales of $28 million, up only 7% sequentially. The Irish company had hoped a faster ramp-up, but the reality of physician education and complex AML choices got in the way. As a result, Jazz dialed back its 2018 forecast for the drug to the range of $115 million to $135 million, but maintained its long-term vision to make Vyxeos the backbone of AML therapy as it expands globally and generates more clinical data.

Lastly, AstraZeneca said Bydureon BCise device was approved in Europe. It’s an improved prefilled pen of the GLP-1 that features a stable-release delivery system. Bydureon sales have suffered from tough in-class competition from Eli Lilly’s Trulicity. The BCise launch in the U.S. only recently helped Bydureon turn in some 6% growth in the second quarter, to $155 million.  For the same quarter, Trulicity posted $780 million.