FDA News Roundup: Otsuka, AstraZeneca, Ipsen, And More

By Anna Rose Welch, Editorial & Community Director, Advancing RNA

What FDA decisions have you missed recently?

Otsuka, Lundbeck Win Approval For Schizophrenia Drug

Otsuka and Lundbeck’s drug Rexulti (brexipiprazole) received approval for adults with major depressive disorder and for adults with schizophrenia. The treatment was investigated in Phase 2 and 3 trials enrolling more than 4,300 patients. The partners are going to co-market the drug which will be released in August.

Veloxis’ Kidney Transplant Treatment Approved

Envarsus XR was approved for patients following a kidney transplant. The drug, taken once daily in tablet form, replaces the need for twice-daily tacrolimus tablets. In two Phase 3 trials and several Phase 2 trials, the drug proved its superiority to Prograf (immediate release tacrolimus), in terms of bioavailability and pharmacokinetic profile. 

Orphan Drug Designations

Cleave Biosciences earned orphan drug designation for CB-5083 for multiple myeloma. The drug inhibits the enzyme p97 which plays several roles in protein homeostasis. There are currently two Phase 1 trials in progress investigating the treatment’s effect in patients with relapsed/refractory multiple myeloma and patients with solid tumors.

Immunovaccine’s ovarian cancer treatment DPX-Survivac was named an orphan drug. The treatment, which targets tumor antigen surviving, is currently indicated for patients with all stages of the disease. In a Phase 1 clinical trial, patients receiving the treatment in combination with low-dose oral cyclophosphamide experienced strong immune responses. 

Newron Pharma won orphan designation for its Rett Syndrome treatment Sarizotan. The treatment aims to improve apnea, hyperventilation, and breath-holds, halting cardio-respiratory complications in women with the disease. The treatment is a 5HT1A agonist and D2 agonist/antagonist that has undergone preclinical testing. In this testing, acute and chronic dosing of Sarizotan led to a 70 to 85 percent reduction in apneas and hyperventilation episodes.

Novogen’s Anisina received orphan drug designation for neuroblastoma. Preclinical studies in animal models of neuroblastoma proved the treatment improved the standard of care microtubule targeting compound, vincristine.

AstraZeneca Gets Green Light For NSCLC Treatment

AstraZeneca’s Iressa for epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer won approval. The tyrosine kinase inhibitor (TKI)targets EGFR exon 19 deletions or exon 21 substitution mutations. Qiagen and AstraZeneca partnered up to develop therascreen as a companion diagnostic test to determine a cancer’s EGFR mutation profile. Data from two studies—the Phase 4 Iressa Follow-Up Measure study and the Iressa Pan-Asia Study — supported the treatment’s approval as a first-line treatment in Caucasian patients. AstraZeneca is also investigating the drug’s potential in immunotherapy combinations for lung cancer.  

Ipsen Injection Receives Approval

Dysport injection, indicated for adults with upper limb spasticity, received approval. In a clinical program enrolling over 600 patients, those receiving the treatment experienced less stiffness, flexing, spasms, and twitching of the elbow, wrist, and finger flexors.