Japan cautiously lifts decades-old bans on somatropin’s use in diabetic patients and interferon beta 1a/1b in pregnant patients, judging benefits can outweigh risks and following requests from doctors and pharma firms to consider patients.

The past few months have seen the European Medicines Agency award its much sought-after priority medicines designation to treatments for Hunter syndrome, bubble boy disease and narcolepsy.

Among other Asia deals, Takeda adds to pair of recent gene therapy tie-ups with a CNS-focused cell therapy partnership with Immusoft; earlier it licensed ex-US rights to JCR’s Hunter syndrome drug.

Denali Therapeutics has reported promising early clinical data in Hunter syndrome for its fusion protein, DNL310, an investigational agent that crosses the blood-brain barrier and is expected to address disease-associated CNS effects; however, some biomarker data disappointed, and there are potential competitors also under development.