Refrigerated Storage

OrfadinSwedish Orphan Biovitrum

Swedish Orphan Biovitrum
2mg, 5mg, 10mg, 20mg capsules; 4mg/ml Oral Suspension

During the shelf life, the patient may store the capsules for a single period of 2 months (for 2 mg capsules) or 3 months (for 5 mg, 10 mg and 20 mg capsules) at a temperature not above 25°C, after which the medicinal product must be discarded.

After first opening, the in-use stability of the 4mg/ml oral suspension is a single period of 2 months at a temperature not above 25°C, after which it must be discarded.

Please also refer to the manufacturer’s product literature for recommended storage information at

Contact Swedish Orphan Biovitrum in all cases where a deviation from the recommended storage conditions has occurred. Refer to the current BNF for company contact details.

Yes - see above
19 November 2020
London MI Service

Medicine Compliance Aid Stability

OrfadinSwedish Orphan Biovitrum Ltd

Swedish Orphan Biovitrum Ltd
Capsules 2mg, 5mg, 10mg
A3 · Amber 3No stability data is available. There are theoretical concerns with use in CAs, which may be mitigated by risk minimisation.
Protect from moisture
Protect from moisture. Should be stored at 2-8 deg C. May remove for a single period of up to 3 months.
29 March 2015

New Medicines

OrfadinMetabolic disease; alkaptonuria (AKU) in adults


Licence extension / variation
Swedish Orphan Biovitrum (Sobi)
Swedish Orphan Biovitrum (Sobi)

Development and Regulatory status

Phase III Clinical Trials
October 2020
Oct 20Licence extension approved in EU [11,12].
Sep 20the EMA has adopted a positive opinion for nitisinone for the treatment of adult patients with alkaptonuria [10].
Jun 20Currently pre-registration in EU [7].
Jan 17Still listed in SOBI pipeline as PIII [5].
Aug 13Granted orphan drug status in EU (EU/3/02/096) in 2002 [1].


Homogentisic acid lowering drug via enzyme inhibition, dosed once daily.
Alkaptonuria (AKU) is a rare disease, which causes severe, early-onset osteoarthritis. AKU patients are born with the disease. However, with the exception of black urine, none of the symptoms present until patients reach their 20s. AKU is a rare disease with an estimated frequency of one in 250,000 to one in 500,000. In the UK, there are currently only 64 patients [3].
Metabolic disease; alkaptonuria (AKU) in adults

Further information

To be confirmed

Trial or other data

Aug 20SONIA 2 (n=138) found daily urinary HGA excretion significantly decreased by 99.7% in nitisinone group vs no treatment (mean ratio of nitisinone/control 0.003, p<0.0001) as was Clinical evaluation Alkaptonuria Severity Score Index (adjusted mean difference –8.6 points, p=0.023) [9].
Jul 20No update to clinical trial registry for SONIA 2 trial has been made [8].
May 18PIII SONIA 2 study is ongoing and expects to complete Feb 20 [6].
Jan 16PIII SONIA 2 study (NCT01916382 ) is ongoing and has finished recruiting pts. Collection of primary outcome data is now expected to complete Feb 16 [4].
Aug 13NCT01916382 (SONIA 2) is an international, multicentre, randomised, evaluator-blind, no-treatment controlled PIII study to assess the efficacy and safety of once daily nitisinone in 140 patients with alkaptonuria after 12 months of treatment, followed by an additional 36 month treatment period. The primary outcome is 24 hour urine homogentisic acid at 1 year. In clusion criteria include age ≥25 years and a diagnosis of AKU) with clinical symptoms. The study starts Nov 13 and is due to complete Apr 18 (primary outcome data collection Apr 15). The study is being run by the University of Liverpool [2].

Evidence based evaluations