dm+d

Unassigned

New Medicines

ZokinvyMetabolic disease

Information

Zokinvy
New molecular entity
Eiger Bio
Eiger Bio

Development and Regulatory status

None
Pre-registration (Filed)
Launched
Yes
Yes
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
To be confirmed

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

Dec 18: Granted PRIME status in EU [9].


Dec 18: Granted breakthrough therapy status in US [8].


Feb 18: Eiger BioPharmaceuticals plans to conduct End of Phase II meeting with the US FDA for hepatitis D virus infections in February 2018.[7]


Apr 15. The FDA has granted Fast Track designation to lonafarnib, for use in combination with ritonavir for the treatment of hepatitis delta virus infection. [3]


Dec 14: Eiger Bio receives orphan designation from the US FDA and EMA for lonafarnib as investigational treatment for Hepatitis Delta Virus (HDV) Infection [1].


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

Trial or other data

Jan 19: PIII study has a primary completion date of April 2021.[10]


Dec 18: Eiger are recruiting to PIII study (NCT03719313) D-LIVR (Delta Liver Improvement and Virologic Response in HDV) to evaluate an all-oral arm of lonafarib (LNF) boosted with ritonavir (RTV) and a combination arm of LNF boosted with RTV combined with pegylated interferon-alfa (PEG IFN-alfa) vs placebo, in HDV-infected patients. A combined primary endpoint of ≥ 2 log10 decline in HDV RNA and ALT normalization at end of 48 weeks of treatment will be used to assess activity [8].
Jan 17: Jan 17: PII NCT02430194 (LOWR-2) is still recruiting patients; the estimated completion date is now Feb 17. PII NCT02430181 (LOWR-1) completed Nov 15, NCT02527707 (LOWR-4) completed Aug 16. Estimated primary completion date of PIIb NCT02968641 [LOWR-5] is December 2018 [6].

Jan 17: Eiger BioPharmaceuticals plan to initiate PIIb LOWR HDV-5 trial to assess the efficacy, safety, pharmacokinetics and pharmacodynamics of lonafarnib with or without ritonavir in patients with chronic HDV infections (EIG-LNF-004; NCT02960360). The open-label, parallel, randomised trial will enrol approximately 60 patients in Mongolia and the US [5].


Nov 16: Eiger BioPharmaceuticals present interim efficacy, adverse events, pharmacodynamics data from the phase II LOWR HDV-2, LOWR HDV-3 and pooled analysis of the LOWR HDV programme in Hepatitis D (Combination therapy) at the American Association for the Study of Liver Diseases Meeting (AASLD-2016). An all-oral optimal combination of lonafarnib and ritonavir that reatined antiviral efficacy and was well-tolerated was identified and the company intends to explore the dose in future trials for longer durations [5].


Jan 16: Eiger are currently recruiting to 3 PIII studies NCT02430181 (LOWR-1), NCT02430194 (LOWR-2) and NCT02527707 (LOWR-4) in combination with ritonavir. Studies should all be complete by Q1 2017 [4].


Dec 14: Eiger are currently recruiting into a PII randomised, double-blind, interventional, pharmacokinetics study (NCT01495585) to test the safety and effectiveness of lonafarnib as a treatment for chronic hepatitis D. Adult pts with HBsAg and HDV RNA in serum, elevated aminotransferases, or moderate-to-severe chronic hepatitis and HDV antigen on liver biopsy will receive either lonafarnib or placebo twice a day for 28 days after which they will stop taking the drug or placebo. They will have regular follow-up visits for up to 6 months after stopping treatment. The primary endpoint will be an improvement in quantitative serum HDV RNA levels after 28 days of lonafarnib therapy. Several secondary endpoints will be measured, including side effects, ALT levels, and symptoms. The study aims to complete during the second quarter of 2016 [2].