U.S. Regulatory News May 2018

Abeona Therapeutics Inc.

FDA granted rare pediatric disease designation for the treatment of CLN1 disease.

Siklos

FDA approved Siklos for paediatric patients, two years of age and older, suffering from sickle cell anaemia.

CSL Behring

Hizentra

FDA approved Hizentra for the treatment of chronic inflammatory demyelinating polyneuropathy.

Cellectar Biosciences

FDA granted orphan drug designation for the treatment of neuroblastoma.

Pedmark

FDA granted fast track designation for the prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma.

Janssen Pharmaceuticals

erdafitinib

FDA granted breakthrough therapy designation for the treatment of urothelial cancer.

Novartis

Tasigna

FDA approved expanded use for the treatment of first- and second-line pediatric patients one year of age or older with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase.

OV101

FDA granted fast track designation for the treatment of Fragile X syndrome. 

Xtandi

FDA granted priority review designation that would expand the indication of the drug to include men with non-metastatic Castration-Resistant Prostate Cancer.

Proteostasis Therapeutics Inc.

PTI-428

FDA granted orphan drug designation and breakthrough therapy designation for the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene as an add-on therapy to approved CFTR modulators or as part of PTI’s proprietary triple combination regimen that includes PTI-808, a potentiator, and PTI-801, a corrector.

Adcetris

FDA approved expanded use for the treatment of adult patients with previously untreated stage III or IV classical Hodgkin lymphoma in combination with chemotherapy.

Sun Pharmaceutical Industries Ltd. 

Ilumya

FDA approved Ilumya for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy.

Tetra Bio-Pharma Inc.

FDA granted orphan drug designation for the treatment of complex regional pain syndrome.

TTI-621

FDA granted orphan drug designation for the treatment of cutaneous T-cell lymphoma.

Zoetis Inc.

FDA approved Robenz and Lincomix as an aid in the prevention of coccidiosis caused by Eimeria mivati, E. brunetti, E. tenella, E. acervulina, E. maxima, and E. necatrix, and for the control of necrotic enteritis caused or complicated by Clostridium spp. or other organisms susceptible to lincomycin in broiler chickens.

Zoetis Inc.