dm+d

Unassigned

New Medicines

ZokinvyMetabolic disease

Information

Zokinvy
New molecular entity
Eiger Bio
Eiger Bio

Development and Regulatory status

None
Recommended for approval (Positive opinion)
Launched
Yes
Yes
May 22Recommended for EU approval under exceptional circumstances by CHMP for “the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation”. Zokinvy will be available as 50 mg and 75 mg hard capsules [12].
Jan 22Ongoing discussions with CHMP primarily focused on additional statistical analyses of clinical data; CHMP opinion expected in first half of 2022 [11]
Jan 21Eiger launched lonafarnib in the US, for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient progeroid laminopathies in patients one year of age and older [10].
Nov 20 US FDA approve Zokinvy for Hutchinson-Gilford Progeria Syndrome and processing-deficient Progeroid Laminopathies. [9]
Aug 20NDA is no longer being reviewed under accelerated assessment programme [8].
May 20Application has been accepted for accelerated assessment [7].
Apr 20EMA accept MAA for lonafarnib to treat Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Progeroid Laminopathies [6].
Jan 20The company anticipates submitting a Marketing Authorisation Application to the EMA in Q1 2020. Also the rolling NDA submission is planned to complete in 1Q 2020. Preparations for commercial launch are underway [5].
Dec 19Eiger initiates rolling submission of NDA with FDA for treatment of progeria and progeroid laminopathies [4].
Nov 19Following positive pre-submission meetings with the EMA and FDA, Eiger announces that it intends to submit a MAA for progeria and progeroid laminopathies to the EMA in Q1 2020 [3].
Dec 18FDA grants Breakthrough Therapy designation for treatment of HGPS and progeroid laminopathies; it had earlier received Rare Paediatric Disease designation [3].
Dec 18Eiger plan to file a NDA in US in 2019 [1].

Category

Farnesyl transferase inhibitor
14 December 2018HGPS is an extremely rare, autosomal dominant, fatal, premature aging syndrome caused by a mutation in the LMNA gene. Approximately 1 in 20 million people have HGPS. Globally, it is estimated that there are 350 to 400 children with the condition [2].
Metabolic disease
Hutchinson-Gilford progeria syndrome (HGPS or Progeria) and progeroid laminopathies
Oral

Further information

Yes

Evidence based evaluations

SarasarHepatitis D infection - in combination with ritonavir

Information

Sarasar
New molecular entity
Eiger Bio
Eiger Bio

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Farnesyl transferase inhibitor
Hepatitis D occurs as a co-infection with HBV, and leads to more severe liver disease. HDV meets criteria for Orphan Designation in the US (< 200,000 people) and Europe (< 5 in 100,000 people). In some countries, e.g. China, Russia, Central Asia, Turkey, Africa, and South America, prevalence may be as high as 40% [1,2].
Hepatitis D infection - in combination with ritonavir
Oral