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What injections can be given orally or via enteral feeding tubes?

6 November 2020This updated Medicines Q&A is a quick reference summary to different types of enteral feeding tubes, in relation to medication issues. Not all enteral feeding…
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Medicine Compliance Aid Stability

AmmonapsImmedica Pharma AB

Immedica Pharma AB
Ammonaps
Tablets 500mg
R2 · Red 2Drug is not suitable for CAs due to theoretical reasons that cannot be mitigated.
Unsuitable
Hygroscopic
7 November 2022

New Medicines

Urea cycle disorders - oral suspension

Information

New formulation
Acer Therapeutics Inc
Acer Therapeutics Inc

Development and Regulatory status

None
None
Not approved
Yes
Jun 22FDA issues a complete response because of an incomplete contract manufacturer inspection. An agency field investigator was unable to inspect a third-party production partner. The FDA has asked for written notification once the facility is ready for inspection. Acer hopes to resubmit an updated new drug application in Q3 22 [3].
Aug 21Filed to the FDA [2].
May 21Acer Therapeutics intends to discuss regulatory submission plans with the EMA and regulatory approval in other territories outside the US, after completion of the NDA filing with the US FDA [2].
Aug 14Has orphan drug status in US [2].

Category

A fully taste-masked, immediate-release formulation of sodium phenylbutyrate, a pro-drug rapidly metabolised to phenylacetate, which then conjugates with glutamine via acetylation to form phenylacetylglutamine (PAG). PAG is excreted by the kidneys, thus providing an alternate vehicle for waste nitrogen excretion and reducing accumulation of ammonia. ACER001 powder formulation microparticles consist of a core centre, a layer of active drug, and a taste-masking coating.
UCDs are rare, affecting 2,100 people in Europe. The US incidence is thought to be one UCD patient for every 35,000 births, representing about 113 new patients each year across all age groups. UCD incidence in Europe is expected to be similar [1].
Urea cycle disorders - oral suspension
Oral

Trial or other data

Dec 20Acer reports full enrolment of its pivotal trial (n=36), that evaluated bioequivalence of sodium phenylbutyrate compared to BUPHENYL® when administered under fed conditions in healthy adults [2].
Feb 20Acer Therapeutics announces completion of part B and successful completion of a pivotal trial, which evaluated the bioavailability and bioequivalence of the optimal formulation of sodium phenylbutyrate, compared with BUPHENYL®. The study showed bioequivalence to BUPHENYL® under fasted conditions, and a significant food effect with sodium phenylbutyrate. Following the completion, the company initiated the non-clinical work and evaluation of long-term product stability. Part B of the trial was a single-centre, open-label, randomised, single-dose crossover trial, which recruited 36 healthy volunteers. The company announced the results from the part B of the trial, which demonstrated that sodium phenylbutyrate was bioequivalent to BUPHENYL® and were within the parameters recommended by the FDA [2].
Oct 19Acer Therapeutics announces that a two part pivotal bioequivalence and bioavailability trial is being conducted in 56 healthy participants for the treatment of UCD in the US. Part A of the study is single-centre, single-blind, randomised, single-dose crossover trial that will evaluate the relative bioavailability of three different oral suspension formulations of sodium phenylbutyrate compared to BUPHENYL® and completed enrolment of 20 healthy adult participants [2].