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When patients undergo transplant procedures, an immunosuppressive drug regimen that prevents rejection of the organ or stem cells can lead to a potentially fatal complication. This week, approved by the European Commission cell therapy to treat this rare disease. The regulatory decision also makes the Atara Biotherapeutics product the first allogeneic cell therapy approved anywhere in the world.

Atara’s product, tabelecleucel, targets a blood cancer called Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+PTLD). In healthy people, T cells keep the Epstein-Barr virus in check. EBV+PTLD develops when a suppressed immune system cannot stop this virus from infecting white blood cells, which then grow uncontrollably. The Thousand Oaks, California-based company’s European marketing authorization for the cell therapy covers the treatment of relapsed or refractory EBV+PTLD in adults and children 2 years of age and older.

Atara cell therapy, a brand of Ebvallo, is made from bioengineered T cells from healthy donors. Unlike first-generation cell therapies—made in a lengthy process that involved collecting a patient’s T cells, engineering them in a lab, and then re-infusing them into the patient—Atara’s allogeneic cell therapy offers patients and clinicians a ready-to-use option .

Ebvallo’s European approval covers those who have received at least one prior therapy, such as Rituxan, a blood cancer drug that is the standard treatment for EBV+PTLD. In solid organ transplant patients, chemotherapy is considered prior therapy. Last year, Atara reached a licensing agreement with French healthcare company Pierre Fabre, which will lead the commercialization of the cell therapy in Europe and some emerging markets. That deal put Atara in line for up to $348 million in milestone payments tied to regulatory and commercial milestones.

Ebvalla has not yet been submitted to the FDA for review, although the company has met with the agency to discuss the requirements for such a submission. Atara said it is looking for a partner to potentially commercialize the cell therapy in the US

The European approval for Ebvallo was one of several notable regulatory developments on both sides of the Atlantic this past week. Here’s a roundup of biotech regulatory news:

— Reform of the FDA’s expedited approval program is part of a broad fiscal 2023 expense account this is making its way through Congress. In addition to giving the FDA more money, the omnibus bill could also allow the agency to require drugmakers to begin confirmatory testing of a drug before it can receive accelerated approval. The legislation also calls for companies to submit “timely reports” at least every six months until the confirmatory study is completed or terminated.

— Actemra, a Roche drug whose approved indications include rheumatoid arthritis, won additional FDA approval as a treatment for Covid-19. The latest regulatory green light for the drug covers hospitalized adults who are being treated with systemic corticosteroids and require breathing assistance from supplemental oxygen, mechanical ventilation or extracorporeal membrane oxygenation (ECMO). The FDA granted emergency use authorization for Actemra for Covid-19 in June 2021. The approval makes the drug, dosed as a single 60-minute infusion, the first monoclonal antibody approved by the agency for the treatment of Covid-19.

— FDA approves first gene therapy for bladder cancer. Ferring Pharmaceuticals’ product, Adstilarin delivers genetic instructions to the patient’s bladder wall cells that cause them to produce large amounts of interferon-alpha 2b, a therapeutic protein with anticancer properties. An earlier application seeking regulatory approval of the gene therapy was rejected in 2020 due to manufacturing issues. Fering said Adstilarin will become available in the second half of next year after the company expands its manufacturing capacity.

— Lynparza, a cancer drug marketed by partners AstraZeneca and Merck, was approved in Europe as first-line treatment for metastatic castration-resistant prostate cancer (mCRPC) in those for whom chemotherapy is unlikely to offer benefit. The European Commission decision covers the use of the drug in combination with the cancer therapy abiraterone, as well as with the steroids prednisone or prednisolone.

Lynparza is what is called a PARP inhibitor. The drug interferes with the PARP enzyme that cancer cells rely on to repair DNA damage. The drug’s FDA approvals cover ovarian, breast, pancreatic and prostate cancers. Under the collaboration agreement, the latest approval for the drug results in a principal payment of $105 million from Merck to AstraZeneca.

— FDA lifted its partial clinical hold on Bluebird Bio’s sickle cell gene therapy tests in patients under 18 years of age. Last year, the agency placed a partial hold on the therapy, lovotibeglogene autotemcel (lovo-cel), after a teenage patient developed anemia after dosing with the experimental treatment.

At the recent annual meeting of the American Society of Hematology, data were presented from an investigation of this case and another in an elderly patient who developed persistent anemia after receiving lovo-cel. The case studies show that both patients have certain alpha-thalassemia, a genetic mutation that can lead to anemia. Bluebird said this specific genotype has since been added to the exclusion criteria for current gene therapy trials.

— Entrada Therapeutics’ plan to begin clinical testing of its experimental treatment for Duchenne muscular dystrophy has failed. The FDA placed a clinical detention on the Boston company’s exploratory application of a new drug, a move that has become increasingly common when the agency stumbles upon new therapeutic modalities.

Entrada is developing a new class of drugs based on a technology platform that enables the delivery of a wide range of therapeutics into cells. Earlier this month, Vertex Pharmaceuticals committed $250 million to get started research alliance using Entrata’s technology to develop drugs for a different muscle disease, myotonic dystrophy type 1.

— Tymlos, Radius Health’s osteoporosis drug for women, is now available approved for men. The FDA expanded the drug’s label to include increasing bone density in men with osteoporosis who are at high risk of fracture. Tymlos, a peptide drug that targets a pathway involved in bone formation, won initial FDA approval in 2017. After eight years as a public company, Radius Health was taken private earlier this year by two private equity firms.

— Vraylar, AbbVie’s blockbuster mental health drug, now has a new indication. The FDA has approved the once-daily capsule for use in addition to antidepressants such as treatment of major depressive disorder. The drug’s previous approvals cover bipolar disorder, schizophrenia and bipolar depression. This year, the drug reported $1.4 billion in revenue in the third quarter, an 18.8% increase compared to the same period in 2021.

Photo: sinonimas, Getty Images

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