Roche company Genentech presented one-year data from FIREFISH Part 2, a pivotal trial of risdiplam in infants one to seven months old with symptomatic Type 1 spinal muscular atrophy (SMA).

Roche company Genentech announced positive top-line results from the pivotal Part 2 of the FIREFISH study looking at risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA).

Novartis AG laid the blame for the manipulation of data behind the company’s $2.1 million gene therapy Zolgensma at the feet of the former executives Brian and Allan Kaspar.

Mallinckrodt Plc tentatively agreed to pay $15.4 million to resolve a U.S. Justice Department probe into how a drugmaker that the company now owns marketed an expensive treatment for a rare infant seizure disorder and multiple sclerosis.

San Diego-based Arcturus Therapeutics Ltd. announced that the company was reassuming 100 percent of its global rights from Germany-based CureVac AG for ARCT-810, a messenger RNA (mRNA) drug to treat OTC deficiency.

Britain’s AstraZeneca said a potential medicine to prevent respiratory syncytial virus (RSV) in babies and infants had been granted special status by U.S. and European regulators, designed to speed up the development of novel and better drugs.

AstraZeneca took another step to refocus on priority drugs by selling U.S. rights to Synagis for infant lung infections to Swedish Orphan Biovitrum for an upfront fee of $1.5 billion.

An FDA advisory panel voted against approving Alkermes Plc’s depression treatment ALKS 5461 in patients with an inadequate response to standard antidepressant therapies.

Privately held pharmaceutical company kaléo announced that the U.S. Food and Drug Administration approved its supplemental New Drug Application for AUVI-Q (epinephrine injection, USP) 0.1 mg.

Biogen’s staggering price tag for the spinal muscular atrophy drug Spinraza is likely to further ignite the controversies of drug pricing.