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Akcea and Ionis' Waylivra nets CHMP approval in ultra-rare familial chylomicronaemia syndrome

Akcea Therapeutics and Ionis Pharmaceuticals have announced that their investigational therapy Waylivra (volanesorsen) has been recommended by the EMA’s Committee for Medicinal Products for Human Use (CHMP) for conditional marketing authorisation as a supplement to diet in genetically confirmed familial chylomicronaemia syndrome (FCS) in adult patients who have not responded adequately to diet and triglyceride lowering therapy and who are at high risk of pancreatitis.

An ultra-rare condition, FCS is caused by impaired function of the enzyme lipoprotein lipase (LPL) and is character

Ionis/Akcea's ultra-rare disease drug rejected by FDA

The FDA has opted to refuse approval to Akcea and Ionis’ Waylivra (volanesorsen) for the treatment of the ultra-rare hereditary condition familial chylomicronemia syndrome (FCS), despite the submission of Phase 3 data from the largest-ever study of the disease.

Both companies’ shares have plummeted on the news: Akcea’s share prices fell 21.5% while Ionis’ dropped by 14%.

Biogen boosts pipeline with $1bn Ionis deal

Somewhat unusually for an early drug development deal, Biogen has put down a serious amount of cash up-front in its $1 billion drug development deal with Ionis Pharmaceuticals.

Perhaps signifying the confidence Biogen has in Ionis’ technology, it was prepared to pay $375 million to secure up to seven drug candidates.

The rest of the deal is made up of Biogen cementing its stake in the company, by acquiring $625 million worth of stock.

AZ licenses kidney drug from Ionis for $330m

AstraZeneca continues to build on its relationship with San Diego-based Ionis Pharmaceuticals, this time by agreeing a licensing deal for a drug to be developed for the treatment of kidney disease.

The press release on the announcement did not divulge many details on what the target area actually is, only specifying that it was a designed to target a genetically associated form of kidney disease.

The candidate that AZ is forking out to license is IONIS-AZ5-2.5RX – with the 2.5 relating to the drug being a “Generation 2.5 antisense drug”.

Top Ten most popular articles on this week

Just 10 days away from Christmas, and big news is still flowing in the industry. Sanofi remains embroiled in scandal over its potentially defective dengue vaccine in the Philippines, and Teva continues to be rocked by negatives as its Chairman announces his immediate departure from the company, while up to 10,000 job losses are announced.

Check out the week's ten most popular articles on now!

Ionis scores huge breakthrough in Huntington’s, Roche licenses drug

Ionis Pharmaceuticals has revealed findings from its Phase 1/2a study into a treatment for Huntington’s disease and the strong data has been met with enthusiasm from all quarters.

The findings from an early stage trial were strong enough for Roche to immediately jump in to license the treatment, parting with $45 million in the process.

The aim of study was objectively to determine safety of the treatment but it was also used to ascertain whether the therapy, known as IONIS-HTTRX, reduced the production of the toxic huntingtin protein.

EMA gives positive opinion on Biogen’s spinal muscular atrophy drug Spinraza

Biogen has revealed that Spinraza (nusinersen), its treatment for spinal muscular atrophy (SMA) , has received a positive opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP).

Novartis and Ionis enter $1bn cardiovascular drug deal

Ionis Pharmaceuticals and one of its wholly-owned subsidiaries Akcea Therapeutics have entered into a partnership with Novartis worth potentially more than $1 billion concerning the exclusive global development and marketing rights for two novel cardiovascular treatments.

Ionis achieves successful Phase 3 trial to treat rare diseases

Ionis Pharmaceutial announced that the results of a Phase 3 trial into its treatment for hypertriglyceridemia met its primary endpoint. The treatment, developed by Ionis’ subsidiary Akcea, showed a significant reduction in triglycerides found in the blood as compared with those who were treated solely by placebo.

AZ secures rights from Ionis for first-ever antisense KRAS cancer drug in $165m deal

AstraZeneca has paid a $28 million milestone payment in its partnership with Ionis Pharmaceuticals over the licensing rights to anticancer treatment AZD4785, the first clinically-developed drug to directly target the KRAS gene regardless of mutation type.

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