dm+d

329482000

Articles

Safety in Lactation: Drugs used in hypoplastic, haemolytic, and renal anaemias

23 September 2020Biosynthetic erythropoietins are almost clinically indistinguishable from the natural form of erythropoietin, which is a normal component of breast-milk. They will not be absorbed from…
Search Articles

Medicine Compliance Aid Stability

FerriproxSwedish Orphan Biovitrum Ltd

Swedish Orphan Biovitrum Ltd
Ferriprox
Tablets f/c 500mg
A2 · Amber 2No stability data is available, the manufacturer does not, or cannot recommend use in CAs but there are no theoretical concerns with the product.
No special precautions for storage
No special storage requirements.
29 March 2015

FerriproxSwedish Orphan Biovitrum Ltd

Swedish Orphan Biovitrum Ltd
Ferriprox
Tablets f/c 1 gram
R2 · Red 2Drug is not suitable for CAs due to theoretical reasons that cannot be mitigated.
Unsuitable
Contains dessicant in pack.
29 March 2015

Lactation Safety Information

No
Deferasirox, Desferrioxamine
No published evidence of safety
Neutropenia reported in adults
18 September 2020

New Medicines

Ferriprox Beta-thalassaemia - enteric-coated gastro-resistant tablet formulation

Information

Ferriprox
New formulation
Chiesi
Chiesi

Development and Regulatory status

Pre-registration (Filed)
Pre-registration (Filed)
Launched
May 20Twice-a-day Ferriprox tablets launched in US [6].
May 20US FDA approved Ferriprox® (deferiprone) twice-a-day tablets via accelerated approval for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate. The new formulation of twice-a-day ferriprox 1000 mg oral tablets eliminates the mid-day dose. Approval is based on a reduction in serum ferritin levels. There are no controlled trials demonstrating a direct treatment benefit, such as improvement in disease-related symptoms, functioning, or increased survival [6].
May 20Approved in the US [5].
Apr 20Currently pre-registration in the EU [3].
Jan 20Chiesi Farmaceutici and Apotex finalise an agreement for the worldwide rights to deferiprone. Once the agreement is approved by the regulatory authorities deferiprone franchise will become a part of Chiesi Group. In addition, a team of 50 employees from ApoPharm (subsidiary of Apotex) will be transferred to Chiesi Group [4].

Category

Chelating agent. Formulated as an enteric coated gastro-resistant tablet for twice daily administration (original film-coated tablet formulation is taken three times a day)
1.5% (80-90 million people) of the world population are carriers of β thalassaemia and 5% are carriers of α thalassaemia. β thalassaemia is prevalent in areas around the Mediterranean, in the Middle East, in Central, South, and Southeast Asia, and in Southern China [1]. It is estimated to affect ~12 per 100,000 of the UK population, although the prevalence in some ethnic groups is substantially greater. There are an estimated 1500 patients in the UK [3].
Beta-thalassaemia - enteric-coated gastro-resistant tablet formulation
Oral

Trial or other data

Dec 19PII TWICE study completes [2].
Mar 19PII TWICE study to assess the safety, tolerability, and acceptability of twice-daily dosing with deferiprone delayed-release (DR) tablets in patients with systemic iron overload who are currently taking deferiprone immediate-release tablets (Ferriprox) three times a day starts (NCT03802916). Ferriprox doses range from 75 milligrams per kilogram of body weight (mg/kg) per day to 100 mg/kg per day. Half the patients in the study will be on a dosage that is closer to the low end of the range, and half will be on a dosage that is closer to the high end. Both groups will be switched for one month to deferiprone DR tablets at approximately the same total daily dosage that they have been taking for Ferriprox. 30 adults will be recruited in the US, Canada, Greece and Italy [2].
Jul 15PI study (NCT02442310) completes [2].
May 15PI study to assess the pharmacokinetics of a new formulation of deferiprone (deferiprone delayed release tablets) under fed and fasting conditions starts (NCT02442310). 20 adults will be recruited at a single centre in Canada. The study is due to complete Jul 15 [2].

Ferriprox Transfusional iron overload due to sickle cell disease or other anaemias - original film-coated tablet formulation

Information

Ferriprox
Licence extension / variation
Chiesi
Chiesi

Development and Regulatory status

Phase III Clinical Trials
None
None
Yes

Category

Chelating agent. Original film-coated tablet formulation is taken three times a day.
Iron excess can occur from as little as 10 transfusions. It is a common problem in transfusion-dependent patients - for example, those with thalassaemia major, sickle cell disease and myelodysplastic syndromes [1].
Transfusional iron overload due to sickle cell disease or other anaemias - original film-coated tablet formulation
Oral

Evidence based evaluations

Ferriprox Transfusional iron overload due to sickle cell disease or other anaemias - gastro-resistant tablet formulation

Information

Ferriprox
Licence extension / variation
Chiesi
Chiesi

Development and Regulatory status

Phase III Clinical Trials
None
Launched
Yes
May 21US FDA approve deferiprone (Ferriprox) for the treatment of transfusional iron overload due to sickle cell disease [4].
Mar 11Granted orphan drug status in EU (EU/3/10/832) [2].

Category

Chelating agent. Formulated as an enteric coated gastro-resistant tablet for twice daily administration (original film-coated tablet formulation is taken three times a day)
Iron excess can occur from as little as 10 transfusions. It is a common problem in transfusion-dependent patients - for example, those with thalassaemia major, sickle cell disease and myelodysplastic syndromes [1].
Transfusional iron overload due to sickle cell disease or other anaemias - gastro-resistant tablet formulation
Intravenous infusion

Trial or other data

Aug 20PIII study (2014-005685-30) started in Sep 15, recruited in the UK, and has prematurely ended. The other PIII study (2013-002181-39) started in Oct 2013 and was recruiting adults and children in the UK; it has also prematurely ended [3].
Aug 20Two PIII studies have been conducted in support of the licence extension for Ferriprox original film-coated tablet - a PIII long-term safety and efficacy study of Ferriprox® for the treatment of transfusional iron overload in patients with sickle cell disease or other anaemias (EudraCT Number: 2014-005685-30) and a PIII study assessing the efficacy and safety of Ferriprox® for the treatment of transfusional iron overload in patients with sickle cell disease or other anaemias (EudraCT Number: 2013-002181-39) [2].

UpkanzPantothenate kinase‐associated neurodegeneration (PKAN) (Hallervorden‐Spatz disease)

Information

Upkanz
New formulation with new indication
Chiesi
Chiesi

Development and Regulatory status

Filing withdrawn
Filing withdrawn
Phase III Clinical Trials
Yes
Sep 20An EMA filing supported by the results of the PIII trial has been withdrawn by company. At the time of withdrawal, there were unresolved issues; in addition, development and marketing of deferiprone were being reassessed following the transfer to Chiesi. The CHMP provisional opinion was that the main study had not clearly shown that the medicine was effective. Therefore, at the time of the withdrawal, the Agency’s opinion was that the benefits of deferiprone in PKAN did not outweigh its risks [7].
Jan 20Chiesi Farmaceutici and Apotex finalise an agreement for the worldwide rights to deferiprone. Once the agreement is approved by the regulatory authorities deferiprone franchise will become a part of Chiesi Group. In addition, a team of 50 employees from ApoPharm (subsidiary of Apotex) will be transferred to Chiesi Group [6].
Aug 18Orphan drug status awarded in EU [5].

Category

Chelating agent
PKAN is the most common type of neurodegeneration with brain iron accumulation, a rare neurodegenerative disorder characterized by progressive extrapyramidal dysfunction (dystonia, rigidity, choreoathetosis), iron accumulation on the brain and axonal spheroids in the central nervous system. Prevalence is estimated at 1-2 per 1,000,000 [1].
Pantothenate kinase‐associated neurodegeneration (PKAN) (Hallervorden‐Spatz disease)
Oral

Trial or other data

Jun 19Results of PIII TIRCON2012V1 (NCT01741532RCT; n=88) found deferiprone was well tolerated, achieved lowering of iron in basal ganglia, and slowed disease progression at 18 months vs. placebo, on Barry-Albright Dystonia scale, although not significantly. Patient Global Impression of Improvement scale was largely unchanged [3].
Jan 17ApoPharma completes a PIII trial that investigated safety and efficacy of deferiprone in patients with PKAN (TIRCON2012V1; NCT01741532). Patients aged 4 years and above received deferiprone 80 mg/mL oral solution, or placebo, twice daily for 18 months. The primary aim was to determine the effects on severity of dystonia; secondary outcomes included pharmacokinetics, tolerability and effects on sleep and quality of life. The trial enrolled 89 patients in the US, Germany Italy and in the UK. ApoPharma initiated the PIII TIRCON-EXT trial in Jun 14 to investigate the safety of long-term deferiprone in patients with PKAN (TIRCON2012V1-EXT; NCT02174848). The trial is enrolling patients that completed the TIRCON2012V1 trial by invitation only [2].